Craig McDonald

Title(s)Professor, Physical Medicine and Rehabilitation
SchoolSchool of Medicine
AddressACC/UCDMC
CA 95817
Phone916-734-2923
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    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Researchers can login to make corrections and additions, or contact us for help. to make corrections and additions.
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    Altmetrics Details PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
    1. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2024 Apr; 23(4):393-403. Mercuri E, Vilchez JJ, Boespflug-Tanguy O, Zaidman CM, Mah JK, Goemans N, Müller-Felber W, Niks EH, Schara-Schmidt U, Bertini E, Comi GP, Mathews KD, Servais L, Vandenborne K, Johannsen J, Messina S, Spinty S, McAdam L, Selby K, Byrne B, Laverty CG, Carroll K, Zardi G, Cazzaniga S, Coceani N, Bettica P, McDonald CM, EPIDYS Study Group. PMID: 38508835.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCTClinical Trials
    2. Survival among patients receiving eteplirsen for up to 8 years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls. Muscle Nerve. 2024 Mar 14. Iff J, Done N, Tuttle E, Zhong Y, Wei F, Darras BT, McDonald CM, Mercuri E, Muntoni F. PMID: 38482981.
      View in: PubMed   Mentions:    Fields:    
    3. Gait Event Detection and Travel Distance Using Waist-Worn Accelerometers across a Range of Speeds: Automated Approach. Sensors (Basel). 2024 Feb 09; 24(4). Ramli AA, Liu X, Berndt K, Chuah CN, Goude E, Kaethler LB, Lopez A, Nicorici A, Owens C, Rodriguez D, Wang J, Aranki D, McDonald CM, Henricson EK. PMID: 38400313.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    4. Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial. Neurology. 2024 Mar 12; 102(5):e208112. Dang UJ, Damsker JM, Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans NM, Haberlová J, Straub V, Mengle-Gaw L, Schwartz BD, Harper A, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster RI, Mcmillan HJ, Kuntz N, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez JJ, Nascimento-Osorio A, Niks EH, De Groot IJM, Katsalouli M, Van Den Anker JN, Ward LM, Leinonen M, D'Alessandro AL, Hoffman EP. PMID: 38335499.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    5. Gait Characterization in Duchenne Muscular Dystrophy (DMD) Using a Single-Sensor Accelerometer: Classical Machine Learning and Deep Learning Approaches. Sensors (Basel). 2024 Feb 08; 24(4). Ramli AA, Liu X, Berndt K, Goude E, Hou J, Kaethler LB, Liu R, Lopez A, Nicorici A, Owens C, Rodriguez D, Wang J, Zhang H, Aranki D, McDonald CM, Henricson EK. PMID: 38400281.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    6. Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy. Pediatr Neurol. 2024 Apr; 153:11-18. Mendell JR, Proud C, Zaidman CM, Mason S, Darton E, Wang S, Wandel C, Murphy AP, Mercuri E, Muntoni F, McDonald CM. PMID: 38306745.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    7. Findings from the Longitudinal CINRG Becker Natural History Study. J Neuromuscul Dis. 2024; 11(1):201-212. Clemens PR, Gordish-Dressman H, Niizawa G, Gorni K, Guglieri M, Connolly AM, Wicklund M, Bertorini T, Mah J, Thangarajh M, Smith EC, Kuntz NL, McDonald CM, Henricson E, Upadhyayula S, Byrne B, Manousakis G, Harper A, Iannaccone S, Dang UJ. PMID: 37980682; PMCID: PMC10789327.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    8. The IAAM LTBP4 Haplotype is Protective Against Dystrophin-Deficient Cardiomyopathy. J Neuromuscul Dis. 2024; 11(2):285-297. Bello L, Sabbatini D, Fusto A, Gorgoglione D, Borin GU, Penzo M, Riguzzi P, Villa M, Vianello S, Calore C, Melacini P, Vio R, Barp A, D'Angelo G, Gandossini S, Politano L, Berardinelli A, Messina S, Vita GL, Pedemonte M, Bruno C, Albamonte E, Sansone V, Baranello G, Masson R, Astrea G, D'Amico A, Bertini E, Pane M, Lucibello S, Mercuri E, Spurney C, Clemens P, Morgenroth L, Gordish-Dressman H, McDonald CM, Hoffman EP, CINRG-DNHS Investigators, Pegoraro E. PMID: 38363615; PMCID: PMC10977446.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    9. Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease. J Neuromuscul Dis. 2024; 11(2):499-523. McDonald C, Camino E, Escandon R, Finkel RS, Fischer R, Flanigan K, Furlong P, Juhasz R, Martin AS, Villa C, Sweeney HL. PMID: 38363616; PMCID: PMC10977441.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    10. Sleep Disordered Breathing in Children with Neuromuscular Disease. Children (Basel). 2023 Oct 11; 10(10). Chidambaram AG, Jhawar S, McDonald CM, Nandalike K. PMID: 37892338; PMCID: PMC10605855.
      View in: PubMed   Mentions:
    11. 269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy - What have we learned? 9-11 December 2022, Hoofddorp, The Netherlands. Neuromuscul Disord. 2023 Nov; 33(11):897-910. Naarding KJ, Stimpson G, Ward SJ, Goemans N, McDonald C, Mercuri E, Muntoni F, 269th ENMC workshop participants. PMID: 37926638.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    12. Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR). Ann Neurol. 2023 11; 94(5):955-968. Zaidman CM, Proud CM, McDonald CM, Lehman KJ, Goedeker NL, Mason S, Murphy AP, Guridi M, Wang S, Reid C, Darton E, Wandel C, Lewis S, Malhotra J, Griffin DA, Potter RA, Rodino-Klapac LR, Mendell JR. PMID: 37539981.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansCells
    13. Correction to: Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis. J Neurol. 2023 Sep; 270(9):4583. Mercuri E, Osorio AN, Muntoni F, Buccella F, Desguerre I, Kirschner J, Tulinius M, de Resende MBD, Morgenroth LP, Gordish-Dressman H, Johnson S, Kristensen A, Werner C, Trifillis P, Henricson EK, McDonald CM, STRIDE and CINRG DNHS investigators. PMID: 37460854; PMCID: PMC10421802.
      View in: PubMed   Mentions:    Fields:    
    14. Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy. Front Cell Dev Biol. 2023; 11:1167762. Mendell JR, Shieh PB, McDonald CM, Sahenk Z, Lehman KJ, Lowes LP, Reash NF, Iammarino MA, Alfano LN, Sabo B, Woods JD, Skura CL, Mao HC, Staudt LA, Griffin DA, Lewis S, Wang S, Potter RA, Singh T, Rodino-Klapac LR. PMID: 37497476; PMCID: PMC10366687.
      View in: PubMed   Mentions: 6  
    15. Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States. J Mark Access Health Policy. 2023; 11(1):2216518. Klimchak AC, Sedita LE, Rodino-Klapac LR, Mendell JR, McDonald CM, Gooch KL, Malone DC. PMID: 37261034; PMCID: PMC10228300.
      View in: PubMed   Mentions:
    16. Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis. J Neurol. 2023 Aug; 270(8):3896-3913. Mercuri E, Osorio AN, Muntoni F, Buccella F, Desguerre I, Kirschner J, Tulinius M, de Resende MBD, Morgenroth LP, Gordish-Dressman H, Johnson S, Kristensen A, Werner C, Trifillis P, Henricson EK, McDonald CM, STRIDE and CINRG DNHS investigators. PMID: 37115359; PMCID: PMC10141820.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    17. Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials. Hum Gene Ther. 2023 05; 34(9-10):404-415. Chamberlain JS, Robb M, Braun S, Brown KJ, Danos O, Ganot A, Gonzalez-Alegre P, Hunter N, McDonald C, Morris C, Tobolowsky M, Wagner KR, Ziolkowski O, Duan D. PMID: 36694468; PMCID: PMC10210223.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    18. Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status. J Comp Eff Res. 2023 04; 12(4):e220190. McDonald CM, Marden JR, Shieh PB, Wong BL, Lane H, Zhang A, Nguyen H, Frean M, Trifillis P, Koladicz K, Signorovitch J. PMID: 36749302; PMCID: PMC10402754.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansCTClinical Trials
    19. DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials. Neurology. 2023 04 11; 100(15):e1540-e1554. Muntoni F, Signorovitch J, Sajeev G, Lane H, Jenkins M, Dieye I, Ward SJ, McDonald C, Goemans N, Niks EH, Wong B, Servais L, Straub V, Guglieri M, de Groot IJM, Chesshyre M, Tian C, Manzur AY, Mercuri E, Aartsma-Rus A, Association Française Contre Les Myopathies; on behalf of Universitaire Ziekenhuizen Leuven Group, P. PMID: 36725339; PMCID: PMC10103111.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    20. Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy1. J Neuromuscul Dis. 2023; 10(1):67-79. McDonald CM, Mayer OH, Hor KN, Miller D, Goemans N, Henricson EK, Marden JR, Freimark J, Lane H, Zhang A, Frean M, Trifillis P, Koladicz K, Signorovitch J, PRO-DMD-01 consortium investigators. PMID: 36565131; PMCID: PMC9881035.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    21. Modeling Early Heterogeneous Rates of Progression in Boys with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2023; 10(3):349-364. Fang Y, McDonald CM, Clemens PR, Gordish HD, Illei K, Hoffman EP, CINRG DNHS and Vamorolone 002/003/LTE Investigators, Dang UJ. PMID: 36806514; PMCID: PMC10200136.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    22. Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study. J Neuromuscul Dis. 2023; 10(3):439-447. Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, McDonald CM, Smith EC, Zaidman CM, Nakagawa T, CINRG DNHS Investigators, Hoffman EP. PMID: 37005891; PMCID: PMC10200237.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansCTClinical Trials
    23. Kinematic changes in gait in boys with Duchenne Muscular Dystrophy: Utility of the Gait Deviation Index, the Gait Profile Score and the Gait Variable Scores. Gait Posture. 2023 02; 100:157-164. Sienko S, Buckon C, Bagley A, Fowler E, Heberer K, Staudt L, Sison-Williamson M, McDonald C, Sussman M. PMID: 36529026.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    24. Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA Neurol. 2022 10 01; 79(10):1005-1014. Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans N, Haberlova J, Straub V, Mengle-Gaw LJ, Schwartz BD, Harper AD, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster R, McMillan HJ, Kuntz NL, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez-Padilla JJ, Nascimento-Osorio A, Niks EH, de Groot IJM, Katsalouli M, James MK, van den Anker J, Damsker JM, Ahmet A, Ward LM, Jaros M, Shale P, Dang UJ, Hoffman EP. PMID: 36036925; PMCID: PMC9425287.
      View in: PubMed   Mentions: 16     Fields:    Translation:Humans
    25. Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial. PLoS One. 2022; 17(8):e0272858. Muntoni F, Guglieri M, Mah JK, Wagner KR, Brandsema JF, Butterfield RJ, McDonald CM, Mayhew AG, Palmer JP, Marraffino S, Charnas L, Mercuri E. PMID: 35998119; PMCID: PMC9397979.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansCTClinical Trials
    26. Influence of β1 Adrenergic Receptor Genotype on Longitudinal Measures of Left Ventricular Ejection Fraction and Responsiveness to ß-Blocker Therapy in Patients With Duchenne Muscular Dystrophy. Clin Med Insights Cardiol. 2022; 16:11795468221116838. Kelley EF, Cross TJ, McDonald CM, Investigators C, Hoffman EP, Spurney CF, Bello L. PMID: 36046180; PMCID: PMC9421016.
      View in: PubMed   Mentions: 1  
    27. Delays in pulmonary decline in eteplirsen-treated patients with Duchenne muscular dystrophy. Muscle Nerve. 2022 09; 66(3):262-269. Iff J, Gerrits C, Zhong Y, Tuttle E, Birk E, Zheng Y, Paul X, Henricson EK, McDonald CM, CINRG-DNHS Investigators. PMID: 35715998.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    28. Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging. BMC Cardiovasc Disord. 2022 06 09; 22(1):260. Batra A, Barnard AM, Lott DJ, Willcocks RJ, Forbes SC, Chakraborty S, Daniels MJ, Arbogast J, Triplett W, Henricson EK, Dayan JG, Schmalfuss C, Sweeney L, Byrne BJ, McDonald CM, Vandenborne K, Walter GA. PMID: 35681116; PMCID: PMC9185987.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    29. Genetic modifiers of upper limb function in Duchenne muscular dystrophy. J Neurol. 2022 Sep; 269(9):4884-4894. Sabbatini D, Fusto A, Vianello S, Villa M, Janik J, D'Angelo G, Diella E, Magri F, Comi GP, Panicucci C, Bruno C, D'Amico A, Bertini E, Astrea G, Battini R, Politano L, Masson R, Baranello G, Previtali SC, Messina S, Vita G, Berardinelli A, Mongini T, Pini A, Pane M, Mercuri E, Hoffman EP, Morgenroth L, Gordish-Dressman H, Duong T, McDonald CM, Bello L, Pegoraro E. PMID: 35513612; PMCID: PMC9363325.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    30. Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA. 2022 04 19; 327(15):1456-1468. Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Speed C, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Griggs RC, FOR-DMD Investigators of the Muscle Study Group, Straub V, van Ruiten H, Childs AM, Ciafaloni E, Shieh PB, Spinty S, Maggi L, Baranello G, Butterfield RJ, Horrocks IA, Roper H, Alhaswani Z, Flanigan KM, Kuntz NL, Manzur A, Darras BT, Kang PB, Morrison L, Krzesniak-Swinarska M, Mah JK, Mongini TE, Ricci F, von der Hagen M, Finkel RS, O'Reardon K, Wicklund M, Kumar A, McDonald CM, Han JJ, Joyce N, Henricson EK, Schara-Schmidt U, Gangfuss A, Wilichowski E, Barohn RJ, Statland JM, Campbell C, Vita G, Vita GL, Howard JF, Hughes I, McMillan HJ, Pegoraro E, Bello L, Burnette WB, Thangarajh M, Chang T. PMID: 35381069; PMCID: PMC8984930.
      View in: PubMed   Mentions: 24     Fields:    Translation:Humans
    31. Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab. J Neurol. 2022 Aug; 269(8):4421-4435. Sherlock SP, Palmer J, Wagner KR, Abdel-Hamid HZ, Bertini E, Tian C, Mah JK, Kostera-Pruszczyk A, Muntoni F, Guglieri M, Brandsema JF, Mercuri E, Butterfield RJ, McDonald CM, Charnas L, Marraffino S. PMID: 35396602; PMCID: PMC9294028.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansCTClinical Trials
    32. Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet. 2022 03 12; 399(10329):1049-1058. McDonald CM, Marbán E, Hendrix S, Hogan N, Ruckdeschel Smith R, Eagle M, Finkel RS, Tian C, Janas J, Harmelink MM, Varadhachary AS, Taylor MD, Hor KN, Mayer OH, Henricson EK, Furlong P, Ascheim DD, Rogy S, Williams P, Marbán L, HOPE-2 Study Group. PMID: 35279258.
      View in: PubMed   Mentions: 17     Fields:    Translation:HumansCTClinical Trials
    33. Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls. Neuromuscul Disord. 2022 04; 32(4):271-283. Muntoni F, Signorovitch J, Sajeev G, Goemans N, Wong B, Tian C, Mercuri E, Done N, Wong H, Moss J, Yao Z, Ward SJ, Manzur A, Servais L, Niks EH, Straub V, de Groot IJ, McDonald C, North Star Clinical Network, PRO-DMD-01 Study, The Association Française contre les Myopathies (AFM). PMID: 35396092.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    34. Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial. JAMA Netw Open. 2022 01 04; 5(1):e2144178. Mah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ, NorthStar UK Network and CINRG DNHS Investigators. PMID: 35076703; PMCID: PMC8790668.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansCTClinical Trials
    35. Influence of β2 adrenergic receptor genotype on longitudinal measures of forced vital capacity in patients with Duchenne muscular dystrophy. Neuromuscul Disord. 2022 02; 32(2):150-158. Kelley EF, Cross TJ, McDonald CM, Hoffman EP, Bello L. PMID: 35115229.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansCells
    36. Knee Strength and Ankle Range of Motion Impacts on Timed Function Tests in Duchenne Muscular Dystrophy: In the Era of Glucocorticoids. J Neuromuscul Dis. 2022; 9(1):147-159. Duong T, Canbek J, Fernandez-Fernandez A, Henricson E, Birkmeier M, Siener C, Tesi Rocha C, McDonald C, Gordish-Dressman H, CINRG Investigators. PMID: 34719507.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    37. A Longitudinal Study of Quantitative Muscle Strength and Functional Motor Ability in Ambulatory Boys with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2022; 9(2):321-334. Buckon CE, Sienko SE, Fowler EG, Bagley AM, Staudt LA, Sison-Williamson M, Heberer KR, McDonald CM, Sussman MD. PMID: 34924398.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    38. A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment. J Neuromuscul Dis. 2022; 9(1):39-52. Mitelman O, Abdel-Hamid HZ, Byrne BJ, Connolly AM, Heydemann P, Proud C, Shieh PB, Wagner KR, Dugar A, Santra S, Signorovitch J, Goemans N, investigators from the LNMRC Natural History study, McDonald CM, investigators from the CINRG Duchenne National History Study, Mercuri E, investigators from The DMD Italian Group, Mendell JR. PMID: 34420980; PMCID: PMC8842766.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    39. Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2022; 9(4):493-501. Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, McDonald CM, Smith EC, Zaidman CM, Nakagawa T, CINRG DNHS Investigators, Hoffman EP. PMID: 35634851; PMCID: PMC9398057.
      View in: PubMed   Mentions: 19     Fields:    Translation:HumansCTClinical Trials
    40. Comparing Deflazacort and Prednisone in Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2022; 9(4):463-476. Biggar WD, Skalsky A, McDonald CM. PMID: 35723111; PMCID: PMC9398085.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    41. Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients. J Comp Eff Res. 2022 02; 11(3):139-155. McDonald CM, Muntoni F, Penematsa V, Jiang J, Kristensen A, Bibbiani F, Goodwin E, Gordish-Dressman H, Morgenroth L, Werner C, Li J, Able R, Trifillis P, Tulinius M, Study 019 investigators. PMID: 34791888; PMCID: PMC8787621.
      View in: PubMed   Mentions: 16     Fields:    Translation:HumansCTClinical Trials
    42. Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy. J Comp Eff Res. 2021 12; 10(18):1337-1347. Shieh PB, Elfring G, Trifillis P, Santos C, Peltz SW, Parsons JA, Apkon S, Darras BT, Campbell C, McDonald CM, Members of the Ataluren Phase IIb Study Group, Members of the Ataluren Phase IIb Study Clinical Evaluator Training Group, Members of the ACT DMD Study Group, Members of the ACT DMD Clinical Evaluator Training Group. PMID: 34693725.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    43. Longitudinal changes in energy cost during walking in boys with Duchenne Muscular Dystrophy (DMD). Gait Posture. 2021 10; 90:301-306. Sienko S, Buckon CE, Bagley A, Staudt L, Fowler E, Heberer K, Sison-Williamson M, McDonald C, Sussman MD. PMID: 34564002.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    44. Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment. Muscle Nerve. 2021 11; 64(5):614-619. McDonald CM, Wei LJ, Flanigan KM, Elfring G, Trifillis P, Muntoni F, ACT DMD Clinical Evaluator Training Group, ACT DMD Study Group. PMID: 34383312; PMCID: PMC9290940.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    45. First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases. Digit Biomark. 2021 May-Aug; 5(2):183-190. Servais L, Camino E, Clement A, McDonald CM, Lukawy J, Lowes LP, Eggenspieler D, Cerreta F, Strijbos P. PMID: 34723071; PMCID: PMC8460979.
      View in: PubMed   Mentions: 19  
    46. Corrigendum to "Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy" [Neuromuscular Disorders, Vol. 30 (6) 2020, 492-502]. Neuromuscul Disord. 2021 Feb; 31(2):167-168. Wagner KR, Abdel-Hamid HZ, Mah JK, Campbell C, Guglieri M, Muntoni F, Takeshima Y, McDonald CM, Kostera-Pruszczyk A, Karachunski P, Butterfield RJ, Mercuri E, Fiorillo C, Bertini ES, Tian C, Statland J, Sadosky AB, Purohit VS, Sherlock SP, Palmer JP, Binks M, Charnas L, Marraffino S, Wong BL. PMID: 33451933.
      View in: PubMed   Mentions: 5     Fields:    
    47. Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls. J Neuromuscul Dis. 2021; 8(4):469-479. Mendell JR, Khan N, Sha N, Eliopoulos H, McDonald CM, Goemans N, Mercuri E, Lowes LP, Alfano LN, Eteplirsen Study Group. PMID: 33523015; PMCID: PMC8385516.
      View in: PubMed   Mentions: 12     Fields:    Translation:Humans
    48. Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial. J Neuromuscul Dis. 2021; 8(6):989-1001. McDonald CM, Shieh PB, Abdel-Hamid HZ, Connolly AM, Ciafaloni E, Wagner KR, Goemans N, Mercuri E, Khan N, Koenig E, Malhotra J, Zhang W, Han B, Mendell JR, the Italian DMD Telethon Registry Study Group, Leuven NMRC Registry Investigators, CINRG Duchenne Na. PMID: 34120909; PMCID: PMC8673535.
      View in: PubMed   Mentions: 31     Fields:    Translation:HumansCTClinical Trials
    49. A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial. J Neuromuscul Dis. 2021; 8(5):769-784. Finkel RS, McDonald CM, Lee Sweeney H, Finanger E, Neil Knierbein E, Wagner KR, Mathews KD, Marks W, Statland J, Nance J, McMillan HJ, McCullagh G, Tian C, Ryan MM, O'Rourke D, Müller-Felber W, Tulinius M, Burnette WB, Nguyen CT, Vijayakumar K, Johannsen J, Phan HC, Eagle M, MacDougall J, Mancini M, Donovan JM, (For the PolarisDMD Study Group). PMID: 34120912; PMCID: PMC8543277.
      View in: PubMed   Mentions: 5     Fields:    Translation:HumansCTClinical Trials
    50. The Minimal Clinical Important Difference (MCID) in Annual Rate of Change of Timed Function Tests in Boys with DMD. J Neuromuscul Dis. 2021; 8(6):939-948. Duong T, Canbek J, Birkmeier M, Nelson L, Siener C, Fernandez-Fernandez A, Henricson E, McDonald CM, Gordish-Dressman H, CINRG-DNHS Investigators. PMID: 34151852; PMCID: PMC8673528.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    51. (-)-Epicatechin induces mitochondrial biogenesis and markers of muscle regeneration in adults with Becker muscular dystrophy. Muscle Nerve. 2021 02; 63(2):239-249. McDonald CM, Ramirez-Sanchez I, Oskarsson B, Joyce N, Aguilar C, Nicorici A, Dayan J, Goude E, Abresch RT, Villarreal F, Ceballos G, Perkins G, Dugar S, Schreiner G, Henricson EK. PMID: 33125736; PMCID: PMC7898288.
      View in: PubMed   Mentions: 18     Fields:    Translation:HumansCells
    52. Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy. J Pers Med. 2020 Nov 19; 10(4). Heier CR, Zhang A, Nguyen NY, Tully CB, Panigrahi A, Gordish-Dressman H, Pandey SN, Guglieri M, Ryan MM, Clemens PR, Thangarajh M, Webster R, Smith EC, Connolly AM, McDonald CM, Karachunski P, Tulinius M, Harper A, Mah JK, Fiorillo AA, Chen YW. PMID: 33228131; PMCID: PMC7711540.
      View in: PubMed   Mentions: 8  
    53. Medical management of muscle weakness in Duchenne muscular dystrophy. PLoS One. 2020; 15(10):e0240687. Rivera SR, Jhamb SK, Abdel-Hamid HZ, Acsadi G, Brandsema J, Ciafaloni E, Darras BT, Iannaccone ST, Konersman CG, Kuntz NL, McDonald CM, Parsons JA, Tesi Rocha C, Zaidman CM, Butterfield RJ, Connolly AM, Mathews KD. PMID: 33075081; PMCID: PMC7571693.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    54. Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. PLoS Med. 2020 09; 17(9):e1003222. Smith EC, Conklin LS, Hoffman EP, Clemens PR, Mah JK, Finkel RS, Guglieri M, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, Kerchner L, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, Gordish-Dressman H, Hagerty L, Dang UJ, Damsker JM, Schwartz BD, Mengle-Gaw LJ, McDonald CM, CINRG VBP15 and DNHS Investigators. PMID: 32956407; PMCID: PMC7505441.
      View in: PubMed   Mentions: 30     Fields:    Translation:HumansCTClinical Trials
    55. Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy. J Comp Eff Res. 2020 10; 9(14):973-984. Campbell C, Barohn RJ, Bertini E, Chabrol B, Comi GP, Darras BT, Finkel RS, Flanigan KM, Goemans N, Iannaccone ST, Jones KJ, Kirschner J, Mah JK, Mathews KD, McDonald CM, Mercuri E, Nevo Y, Péréon Y, Renfroe JB, Ryan MM, Sampson JB, Schara U, Sejersen T, Selby K, Tulinius M, Vílchez JJ, Voit T, Wei LJ, Wong BL, Elfring G, Souza M, McIntosh J, Trifillis P, Peltz SW, Muntoni F, PTC124-GD-007-DMD Study Group, ACT DMD Study Group, Clinical Evaluator Training Groups. PMID: 32851872.
      View in: PubMed   Mentions: 22     Fields:    Translation:Humans
    56. Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial. JAMA Neurol. 2020 08 01; 77(8):982-991. Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP, CINRG DNHS Investigators. PMID: 32453377; PMCID: PMC7251505.
      View in: PubMed   Mentions: 101     Fields:    Translation:HumansCTClinical Trials
    57. The CINRG Becker Natural History Study: Baseline characteristics. Muscle Nerve. 2020 09; 62(3):369-376. Clemens PR, Niizawa G, Feng J, Florence J, D'Alessandro AS, Morgenroth LP, Gorni K, Guglieri M, Connolly A, Wicklund M, Bertorini T, Mah JK, Thangarajh M, Smith E, Kuntz N, McDonald CM, Henricson EK, Upadhyayula S, Byrne B, Manousakis G, Harper A, Bravver E, Iannaccone S, Spurney C, Cnaan A, Gordish-Dressman H, CINRG BNHS Investigators. PMID: 32564389; PMCID: PMC7520226.
      View in: PubMed   Mentions: 11     Fields:    Translation:Humans
    58. Suitability of external controls for drug evaluation in Duchenne muscular dystrophy. Neurology. 2020 09 08; 95(10):e1381-e1391. Goemans N, Signorovitch J, Sajeev G, Yao Z, Gordish-Dressman H, McDonald CM, Vandenborne K, Miller D, Ward SJ, Mercuri E, investigators from PRO-DMD-01 Study, CINRG DNHS, ImagingDMD, and the DMD Italian Group. PMID: 32611643; PMCID: PMC7538219.
      View in: PubMed   Mentions: 18     Fields:    Translation:Humans
    59. Seven-Year Experience From the National Institute of Neurological Disorders and Stroke-Supported Network for Excellence in Neuroscience Clinical Trials. JAMA Neurol. 2020 06 01; 77(6):755-763. Cudkowicz M, Chase MK, Coffey CS, Ecklund DJ, Thornell BJ, Lungu C, Mahoney K, Gutmann L, Shefner JM, Staley KJ, Bosch M, Foster E, Long JD, Bayman EO, Torner J, Yankey J, Peters R, Huff T, Conwit RA, NeuroNEXT Clinical Study Sites, Shinnar S, Patch D, Darras BT, Ellis A, Packer RJ, Marder KS, Chiriboga CA, Henchcliffe C, Moran JA, Nikolov B, Factor SA, Seeley C, Greenberg SM, Amato AA, DeGregorio S, Simuni T, Ward T, Kissel JT, Kolb SJ, Bartlett A, Quinn JF, Keith K, Levine SR, Gilles N, Coyle PK, Lamb J, Wolfe GI, Crumlish A, Mejico L, Iqbal MM, Bowen JD, Tongco C, Nabors LB, Bashir K, Benge M, McDonald CM, Henricson EK, Oskarsson B, Dobkin BH, Canamar C, Glauser TA, Woo D, Molloy A, Clark P, Vollmer TL, Stein AJ, Barohn RJ, Dimachkie MM, Le Pichon JB, Benatar MG, Steele J, Wechsler L, Clemens PR, Amity C, Holloway RG, Annis C, Goldberg MP, Andersen M, Iannaccone ST, Smith AG, Singleton JR, Doudova M, Haley EC, Quigg MS, Lowenhaupt S, Malow BA, Adkins K, Clifford DB, Teshome MA, Connolly N. PMID: 32202612; PMCID: PMC7483960.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    60. Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy. Neuromuscul Disord. 2020 06; 30(6):492-502. Wagner KR, Abdel-Hamid HZ, Mah JK, Campbell C, Guglieri M, Muntoni F, Takeshima Y, McDonald CM, Kostera-Pruszczyk A, Karachunski P, Butterfield RJ, Mercuri E, Fiorillo C, Bertini ES, Tian C, Statland J, Sadosky AB, Purohit VS, Sherlock SP, Palmer JP, Binks M, Charnas L, Marraffino S, Wong BL. PMID: 32522498.
      View in: PubMed   Mentions: 26     Fields:    Translation:HumansCTClinical Trials
    61. The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID-19 pandemic. Muscle Nerve. 2020 07; 62(1):41-45. Veerapandiyan A, Wagner KR, Apkon S, McDonald CM, Mathews KD, Parsons JA, Wong BL, Eichinger K, Shieh PB, Butterfield RJ, Rao VK, Smith EC, Proud CM, Connolly AM, Ciafaloni E. PMID: 32329920; PMCID: PMC7264600.
      View in: PubMed   Mentions: 36     Fields:    Translation:HumansCellsPHPublic Health
    62. Genetic modifiers of respiratory function in Duchenne muscular dystrophy. Ann Clin Transl Neurol. 2020 05; 7(5):786-798. Bello L, D'Angelo G, Villa M, Fusto A, Vianello S, Merlo B, Sabbatini D, Barp A, Gandossini S, Magri F, Comi GP, Pedemonte M, Tacchetti P, Lanzillotta V, Trucco F, D'Amico A, Bertini E, Astrea G, Politano L, Masson R, Baranello G, Albamonte E, De Mattia E, Rao F, Sansone VA, Previtali S, Messina S, Vita GL, Berardinelli A, Mongini T, Pini A, Pane M, Mercuri E, Vianello A, Bruno C, Hoffman EP, Morgenroth L, Gordish-Dressman H, McDonald CM, CINRG-DNHS Investigators, Pegoraro E. PMID: 32343055; PMCID: PMC7261745.
      View in: PubMed   Mentions: 20     Fields:    Translation:Humans
    63. Conference report on contractures in musculoskeletal and neurological conditions. Muscle Nerve. 2020 06; 61(6):740-744. Nuckolls GH, Kinnett K, Dayanidhi S, Domenighetti AA, Duong T, Hathout Y, Lawlor MW, Lee SSM, Magnusson SP, McDonald CM, McNally EM, Miller NF, Olwin BB, Raghavan P, Roberts TJ, Rutkove SB, Sarwark JF, Senesac CR, Vogel LF, Walter GA, Willcocks RJ, Rymer WZ, Lieber RL. PMID: 32108365; PMCID: PMC7229996.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    64. Assessment of Treatment Effect With Multiple Outcomes in 2 Clinical Trials of Patients With Duchenne Muscular Dystrophy. JAMA Netw Open. 2020 02 05; 3(2):e1921306. Li D, McDonald CM, Elfring GL, Souza M, McIntosh J, Kim DH, Wei LJ. PMID: 32058550; PMCID: PMC7202370.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    65. Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study. J Comp Eff Res. 2020 04; 9(5):341-360. Mercuri E, Muntoni F, Osorio AN, Tulinius M, Buccella F, Morgenroth LP, Gordish-Dressman H, Jiang J, Trifillis P, Zhu J, Kristensen A, Santos CL, Henricson EK, McDonald CM, Desguerre I, STRIDE, CINRG Duchenne Natural History Investigators. PMID: 31997646; PMCID: PMC7610147.
      View in: PubMed   Mentions: 36     Fields:    Translation:Humans
    66. TCTEX1D1 is a genetic modifier of disease progression in Duchenne muscular dystrophy. Eur J Hum Genet. 2020 06; 28(6):815-825. Spitali P, Zaharieva I, Bohringer S, Hiller M, Chaouch A, Roos A, Scotton C, Claustres M, Bello L, McDonald CM, Hoffman EP, CINRG Investigators, Koeks Z, Eka Suchiman H, Cirak S, Scoto M, Reza M, 't Hoen PAC, Niks EH, Tuffery-Giraud S, Lochmüller H, Ferlini A, Muntoni F, Aartsma-Rus A. PMID: 31896777; PMCID: PMC7253478.
      View in: PubMed   Mentions: 18     Fields:    Translation:Humans
    67. Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials. Muscle Nerve. 2020 01; 61(1):26-35. McDonald CM, Sajeev G, Yao Z, McDonnell E, Elfring G, Souza M, Peltz SW, Darras BT, Shieh PB, Cox DA, Landry J, Signorovitch J, ACT DMD Study Group and the Tadalafil DMD Study Group. PMID: 31599456; PMCID: PMC6973289.
      View in: PubMed   Mentions: 23     Fields:    Translation:Humans
    68. Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy. Neuromuscul Disord. 2020 01; 30(1):5-16. Servais L, Straathof CSM, Schara U, Klein A, Leinonen M, Hasham S, Meier T, De Waele L, Gordish-Dressman H, McDonald CM, Mayer OH, Voit T, Mercuri E, Buyse GM, SYROS and CINRG DNHS Investigators. PMID: 31813614.
      View in: PubMed   Mentions: 19     Fields:    Translation:HumansCTClinical Trials
    69. Influence of β2 adrenergic receptor genotype on risk of nocturnal ventilation in patients with Duchenne muscular dystrophy. Respir Res. 2019 Oct 16; 20(1):221. Kelley EF, Cross TJ, Snyder EM, McDonald CM, CINRG Investigators, Hoffman EP, Bello L. PMID: 31619245; PMCID: PMC6796481.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    70. Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Neurology. 2019 09 24; 93(13):e1312-e1323. Hoffman EP, Schwartz BD, Mengle-Gaw LJ, Smith EC, Castro D, Mah JK, McDonald CM, Kuntz NL, Finkel RS, Guglieri M, Bushby K, Tulinius M, Nevo Y, Ryan MM, Webster R, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Siener C, Jaros M, Shale P, McCall JM, Nagaraju K, van den Anker J, Conklin LS, Cnaan A, Gordish-Dressman H, Damsker JM, Clemens PR, Cooperative International Neuromuscular Research Group. PMID: 31451516; PMCID: PMC7011869.
      View in: PubMed   Mentions: 40     Fields:    Translation:HumansCTClinical Trials
    71. Disease-specific and glucocorticoid-responsive serum biomarkers for Duchenne Muscular Dystrophy. Sci Rep. 2019 08 21; 9(1):12167. Hathout Y, Liang C, Ogundele M, Xu G, Tawalbeh SM, Dang UJ, Hoffman EP, Gordish-Dressman H, Conklin LS, van den Anker JN, Clemens PR, Mah JK, Henricson E, McDonald C. PMID: 31434957; PMCID: PMC6704115.
      View in: PubMed   Mentions: 24     Fields:    Translation:Humans
    72. Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy. J Pharmacokinet Pharmacodyn. 2019 10; 46(5):441-455. Conrado DJ, Larkindale J, Berg A, Hill M, Burton J, Abrams KR, Abresch RT, Bronson A, Chapman D, Crowther M, Duong T, Gordish-Dressman H, Harnisch L, Henricson E, Kim S, McDonald CM, Schmidt S, Vong C, Wang X, Wong BL, Yong F, Romero K, Duchenne Muscular Dystrophy Regulatory Science Consortium (D-RSC). PMID: 31127458.
      View in: PubMed   Mentions: 11     Fields:    Translation:Humans
    73. Longitudinal study of upper extremity reachable workspace in fascioscapulohumeral muscular dystrophy. Neuromuscul Disord. 2019 07; 29(7):503-513. Hatch MN, Kim K, Kurillo G, Nicorici A, McDonald CM, Han JJ. PMID: 31345604; PMCID: PMC6996236.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    74. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 06; 59(6):650-657. Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network. PMID: 30706490.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    75. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6(2):213-225. Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators. PMID: 30856119; PMCID: PMC6598025.
      View in: PubMed   Mentions: 39     Fields:    Translation:Humans
    76. Timed function tests have withstood the test of time as clinically meaningful and responsive endpoints in duchenne muscular dystrophy. Muscle Nerve. 2018 11; 58(5):614-617. McDonald CM. PMID: 30192014.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    77. Neurodevelopmental Needs in Young Boys with Duchenne Muscular Dystrophy (DMD): Observations from the Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DNHS). PLoS Curr. 2018 10 17; 10. Thangarajh M, Spurney CF, Gordish-Dressman H, Clemens PR, Hoffman EP, McDonald CM, Henricson EK, CINRG Investigators. PMID: 30443431; PMCID: PMC6209412.
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    78. Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial. Muscle Nerve. 2018 11; 58(5):639-645. Shieh PB, Mcintosh J, Jin F, Souza M, Elfring G, Narayanan S, Trifillis P, Peltz SW, Mcdonald CM, Darras BT, THE ACT DMD STUDY GROUP. PMID: 30028519; PMCID: PMC6767037.
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    79. Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug. Pharmacol Res. 2018 10; 136:140-150. Conklin LS, Damsker JM, Hoffman EP, Jusko WJ, Mavroudis PD, Schwartz BD, Mengle-Gaw LJ, Smith EC, Mah JK, Guglieri M, Nevo Y, Kuntz N, McDonald CM, Tulinius M, Ryan MM, Webster R, Castro D, Finkel RS, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, McCall JM, Hathout Y, Nagaraju K, van den Anker J, Ward LM, Ahmet A, Cornish MR, Clemens PR. PMID: 30219580; PMCID: PMC6218284.
      View in: PubMed   Mentions: 40     Fields:    Translation:HumansCTClinical Trials
    80. Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids. Neuromuscul Disord. 2018 11; 28(11):897-909. McDonald CM, Gordish-Dressman H, Henricson EK, Duong T, Joyce NC, Jhawar S, Leinonen M, Hsu F, Connolly AM, Cnaan A, Abresch RT, CINRG investigators for PubMed. PMID: 30336970.
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    81. Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA! Contemp Clin Trials Commun. 2018 Sep; 11:113-119. Bartlett A, Kolb SJ, Kingsley A, Swoboda KJ, Reyna SP, Sakonju A, Darras BT, Shell R, Kuntz N, Castro D, Iannaccone ST, Parsons J, Connolly AM, Chiriboga CA, McDonald C, Burnette WB, Werner K, Thangarajh M, Shieh PB, Finanger E, Coffey CS, Yankey JW, Cudkowicz ME, McGovern MM, McNeil DE, Arnold WD, Kissel JT, NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators. PMID: 30094386; PMCID: PMC6072892.
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    82. Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy. Ann Clin Transl Neurol. 2018 Aug; 5(8):913-926. McDonald CM, Wong B, Flanigan KM, Wilson R, de Kimpe S, Lourbakos A, Lin Z, Campion G, DEMAND V study group. PMID: 30128316; PMCID: PMC6093847.
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    83. A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial. Trials. 2018 May 10; 19(1):291. Crow RA, Hart KA, McDermott MP, Tawil R, Martens WB, Herr BE, McColl E, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Hirtz D, Lochmuller H, Straub V, Ciafaloni E, Shieh PB, Spinty S, Childs AM, Manzur AY, Morandi L, Butterfield RJ, Horrocks I, Roper H, Flanigan KM, Kuntz NL, Mah JK, Morrison L, Darras BT, von der Hagen M, Schara U, Wilichowski E, Mongini T, McDonald CM, Vita G, Barohn RJ, Finkel RS, Wicklund M, McMillan HJ, Hughes I, Pegoraro E, Bryan Burnette W, Howard JF, Thangarajh M, Campbell C, Griggs RC, Bushby K, Guglieri M. PMID: 29793540; PMCID: PMC5968578.
      View in: PubMed   Mentions: 16     Fields:    Translation:Humans
    84. Evidence-based care in Duchenne muscular dystrophy. Lancet Neurol. 2018 05; 17(5):389-391. McDonald CM, Mercuri E. PMID: 29656735.
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    85. A multinational study on motor function in early-onset FSHD. Neurology. 2018 04 10; 90(15):e1333-e1338. Mah JK, Feng J, Jacobs MB, Duong T, Carroll K, de Valle K, Carty CL, Morgenroth LP, Guglieri M, Ryan MM, Clemens PR, Thangarajh M, Webster R, Smith E, Connolly AM, McDonald CM, Karachunski P, Tulinius M, Harper A, Cnaan A, Chen YW, Cooperative International Neuromuscular Research Group (CINRG) Investigators. PMID: 29540582; PMCID: PMC5894929.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansCells
    86. Mexiletine for muscle cramps in amyotrophic lateral sclerosis: A randomized, double-blind crossover trial. Muscle Nerve. 2018 Mar 06. Oskarsson B, Moore D, Mozaffar T, Ravits J, Wiedau-Pazos M, Parziale N, Joyce NC, Mandeville R, Goyal N, Cudkowicz ME, Weiss M, Miller RG, McDonald CM. PMID: 29510461; PMCID: PMC6126993.
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    87. A presynaptic congenital myasthenic syndrome attributed to a homozygous sequence variant in LAMA5. Ann N Y Acad Sci. 2018 02; 1413(1):119-125. Maselli RA, Arredondo J, Vázquez J, Chong JX, Bamshad MJ, Nickerson DA, Lara M, Ng F, Lo VL, Pytel P, McDonald CM. PMID: 29377152; PMCID: PMC6252105.
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    88. Home-Based Monitoring of Pulmonary Function in Patients with Duchenne Muscular Dystroph. J Neuromuscul Dis. 2018; 5(4):419-430. Buyse GM, Rummey C, Meier T, Leinonen M, Voit T, McDonald CM, Mayer OH. PMID: 30282375; PMCID: PMC6218142.
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    89. Natural history of infantile-onset spinal muscular atrophy. Ann Neurol. 2017 Dec; 82(6):883-891. Kolb SJ, Coffey CS, Yankey JW, Krosschell K, Arnold WD, Rutkove SB, Swoboda KJ, Reyna SP, Sakonju A, Darras BT, Shell R, Kuntz N, Castro D, Parsons J, Connolly AM, Chiriboga CA, McDonald C, Burnette WB, Werner K, Thangarajh M, Shieh PB, Finanger E, Cudkowicz ME, McGovern MM, McNeil DE, Finkel R, Iannaccone ST, Kaye E, Kingsley A, Renusch SR, McGovern VL, Wang X, Zaworski PG, Prior TW, Burghes AHM, Bartlett A, Kissel JT, NeuroNEXT Clinical Trial Network on behalf of the NN101 SMA Biomarker Investigators. PMID: 29149772; PMCID: PMC5776712.
      View in: PubMed   Mentions: 120     Fields:    Translation:Humans
    90. A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy. Neuromuscul Disord. 2018 01; 28(1):4-15. Goemans N, Mercuri E, Belousova E, Komaki H, Dubrovsky A, McDonald CM, Kraus JE, Lourbakos A, Lin Z, Campion G, Wang SX, Campbell C, DEMAND III study group. PMID: 29203355.
      View in: PubMed   Mentions: 59     Fields:    Translation:HumansCTClinical Trials
    91. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study. Lancet. 2018 02 03; 391(10119):451-461. McDonald CM, Henricson EK, Abresch RT, Duong T, Joyce NC, Hu F, Clemens PR, Hoffman EP, Cnaan A, Gordish-Dressman H, CINRG Investigators. PMID: 29174484.
      View in: PubMed   Mentions: 180     Fields:    Translation:HumansCTClinical Trials
    92. A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy. Neurology. 2017 Oct 24; 89(17):1811-1820. Victor RG, Sweeney HL, Finkel R, McDonald CM, Byrne B, Eagle M, Goemans N, Vandenborne K, Dubrovsky AL, Topaloglu H, Miceli MC, Furlong P, Landry J, Elashoff R, Cox D, Tadalafil DMD Study Group. PMID: 28972192; PMCID: PMC5664308.
      View in: PubMed   Mentions: 30     Fields:    Translation:HumansCTClinical Trials
    93. Interleukin 1 Receptor-Like 1 Protein (ST2) is a Potential Biomarker for Cardiomyopathy in Duchenne Muscular Dystrophy. Pediatr Cardiol. 2017 Dec; 38(8):1606-1612. Anderson J, Seol H, Gordish-Dressman H, Hathout Y, Spurney CF, CINRG Investigators. PMID: 28821969; PMCID: PMC6317901.
      View in: PubMed   Mentions: 9     Fields:    Translation:Humans
    94. Pulmonary Endpoints in Duchenne Muscular Dystrophy. A Workshop Summary. Am J Respir Crit Care Med. 2017 08 15; 196(4):512-519. Finder J, Mayer OH, Sheehan D, Sawnani H, Abresch RT, Benditt J, Birnkrant DJ, Duong T, Henricson E, Kinnett K, McDonald CM, Connolly AM. PMID: 28636407.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    95. Longitudinal community walking activity in Duchenne muscular dystrophy. Muscle Nerve. 2018 03; 57(3):401-406. Fowler EG, Staudt LA, Heberer KR, Sienko SE, Buckon CE, Bagley AM, Sussman MD, McDonald CM. PMID: 28692198.
      View in: PubMed   Mentions: 16     Fields:    Translation:Humans
    96. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2017 09 23; 390(10101):1489-1498. McDonald CM, Campbell C, Torricelli RE, Finkel RS, Flanigan KM, Goemans N, Heydemann P, Kaminska A, Kirschner J, Muntoni F, Osorio AN, Schara U, Sejersen T, Shieh PB, Sweeney HL, Topaloglu H, Tulinius M, Vilchez JJ, Voit T, Wong B, Elfring G, Kroger H, Luo X, McIntosh J, Ong T, Riebling P, Souza M, Spiegel RJ, Peltz SW, Mercuri E, Clinical Evaluator Training Group, ACT DMD Study Group. PMID: 28728956.
      View in: PubMed   Mentions: 194     Fields:    Translation:HumansCTClinical Trials
    97. The Direct Cost of Managing a Rare Disease: Assessing Medical and Pharmacy Costs Associated with Duchenne Muscular Dystrophy in the United States. J Manag Care Spec Pharm. 2017 Jun; 23(6):633-641. Thayer S, Bell C, McDonald CM. PMID: 28530521; PMCID: PMC10398014.
      View in: PubMed   Mentions: 16     Fields:    Translation:Humans
    98. Presynaptic congenital myasthenic syndrome with a homozygous sequence variant in LAMA5 combines myopia, facial tics, and failure of neuromuscular transmission. Am J Med Genet A. 2017 Aug; 173(8):2240-2245. Maselli RA, Arredondo J, Vázquez J, Chong JX, University of Washington Center for Mendelian Genomics, Bamshad MJ, Nickerson DA, Lara M, Ng F, Lo VL, Pytel P, McDonald CM. PMID: 28544784; PMCID: PMC5541137.
      View in: PubMed   Mentions: 17     Fields:    Translation:Humans
    99. In memoriam: William M. Fowler Jr, MD. Muscle & Nerve. 2017 May 1; 55(5):617-618. McDonald MC, Carter CG. .
      View in: Publisher Site   Mentions:
    100. Developing standardized corticosteroid treatment for Duchenne muscular dystrophy. Contemp Clin Trials. 2017 07; 58:34-39. Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Hirtz D, Shieh PB, Straub V, Childs AM, Ciafaloni E, Butterfield RJ, Horrocks I, Spinty S, Flanigan KM, Kuntz NL, Baranello G, Roper H, Morrison L, Mah JK, Manzur AY, McDonald CM, Schara U, von der Hagen M, Barohn RJ, Campbell C, Darras BT, Finkel RS, Vita G, Hughes I, Mongini T, Pegoraro E, Wicklund M, Wilichowski E, Bryan Burnette W, Howard JF, McMillan HJ, Thangarajh M, Griggs RC. PMID: 28450193; PMCID: PMC6279424.
      View in: PubMed   Mentions: 33     Fields:    Translation:Humans
    101. William M. Fowler, Jr, MD, 1926-2017. PM R. 2017 05; 9(5):540-541. Carter GT, McDonald CM. PMID: 28347900.
      View in: PubMed   Mentions:    Fields:    
    102. Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA. Neuromuscul Disord. 2017 07; 27(7):693-701. Hoffman EP, Workshop Participants, TREAT-NMD Alliance. PMID: 28434909.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    103. Duchenne Regulatory Science Consortium Meeting on Disease Progression Modeling for Duchenne Muscular Dystrophy. PLoS Curr. 2017 Jan 12; 9. Larkindale J, Abresch R, Aviles E, Bronson A, Chin J, Furlong P, Gordish-Dressman H, Habeeb-Louks E, Henricson E, Kroger H, Lynn C, Lynn S, Martin D, Nuckolls G, Rooney W, Romero K, Sweeney L, Vandenborne K, Walter G, Wolff J, Wong B, McDonald CM, Duchenne Regulatory Science Consortium Imaging-Dmd Consortium And The Cinrg Investigators MO. PMID: 28228973; PMCID: PMC5300692.
      View in: PubMed   Mentions: 4  
    104. Characterization of pulmonary function in 10-18 year old patients with Duchenne muscular dystrophy. Neuromuscul Disord. 2017 Apr; 27(4):307-314. Meier T, Rummey C, Leinonen M, Spagnolo P, Mayer OH, Buyse GM, DELOS Study Group. PMID: 28189481.
      View in: PubMed   Mentions: 13     Fields:    Translation:Humans
    105. Advances in Pulmonary Care in Duchenne Muscular Dystrophy. US Neurology. 2017 Jan 1; 13(01):35. Mayer MO, Henricson HE, McDonald MC, Buyse BG. .
      View in: Publisher Site   Mentions:
    106. Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy. Am J Hum Genet. 2016 Nov 03; 99(5):1163-1171. Bello L, Flanigan KM, Weiss RB, United Dystrophinopathy Project, Spitali P, Aartsma-Rus A, Muntoni F, Zaharieva I, Ferlini A, Mercuri E, Tuffery-Giraud S, Claustres M, Straub V, Lochmüller H, Barp A, Vianello S, Pegoraro E, Punetha J, Gordish-Dressman H, Giri M, McDonald CM, Hoffman EP, Cooperative International Neuromuscular Research Group. PMID: 27745838; PMCID: PMC5097949.
      View in: PubMed   Mentions: 41     Fields:    Translation:Humans
    107. Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD Upper Limb PROM. Dev Med Child Neurol. 2017 Feb; 59(2):224-231. Klingels K, Mayhew AG, Mazzone ES, Duong T, Decostre V, Werlauff U, Vroom E, Mercuri E, Goemans NM, Upper Limb Clinical Outcome Group. PMID: 27671699.
      View in: PubMed   Mentions: 15     Fields:    Translation:Humans
    108. Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy. Pediatr Pulmonol. 2017 04; 52(4):508-515. Buyse GM, Voit T, Schara U, Straathof CS, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, Rummey C, Leinonen M, Mayer OH, Spagnolo P, Meier T, McDonald CM, DELOS Study Group. PMID: 27571420; PMCID: PMC5396356.
      View in: PubMed   Mentions: 16     Fields:    Translation:HumansCTClinical Trials
    109. Serum pharmacodynamic biomarkers for chronic corticosteroid treatment of children. Sci Rep. 2016 08 17; 6:31727. Hathout Y, Conklin LS, Seol H, Gordish-Dressman H, Brown KJ, Morgenroth LP, Nagaraju K, Heier CR, Damsker JM, van den Anker JN, Henricson E, Clemens PR, Mah JK, McDonald C, Hoffman EP. PMID: 27530235; PMCID: PMC4987691.
      View in: PubMed   Mentions: 26     Fields:    Translation:Humans
    110. Correction: Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study. PLoS One. 2016; 11(7):e0159895. Boca SM, Nishida M, Harris M, Rao S, Cheema AK, Gill K, Wang D, An L, Gauba R, Seol H, Morgenroth LP, Henricson E, McDonald C, Mah JK, Clemens PR, Hoffman EP, Hathout Y, Madhavan S. PMID: 27434074; PMCID: PMC4951084.
      View in: PubMed   Mentions: 7     Fields:    
    111. Can Quantitative Muscle Strength and Functional Motor Ability Differentiate the Influence of Age and Corticosteroids in Ambulatory Boys with Duchenne Muscular Dystrophy? PLoS Curr. 2016 Jul 08; 8. Buckon C, Sienko S, Bagley A, Sison-Williamson M, Fowler E, Staudt L, Heberer K, McDonald CM, Sussman M. PMID: 27500011; PMCID: PMC4956479.
      View in: PubMed   Mentions: 6  
    112. DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study. Neurology. 2016 07 26; 87(4):401-9. Bello L, Morgenroth LP, Gordish-Dressman H, Hoffman EP, McDonald CM, Cirak S, CINRG investigators. PMID: 27343068; PMCID: PMC4977110.
      View in: PubMed   Mentions: 75     Fields:    Translation:Humans
    113. Prednisone and Deflazacort in Duchenne Muscular Dystrophy: Do They Play a Different Role in Child Behavior and Perceived Quality of Life? PLoS Curr. 2016 Jun 17; 8. Sienko S, Buckon C, Fowler E, Bagley A, Staudt L, Sison-Williamson M, Zebracki K, McDonald CM, Sussman M. PMID: 27525172; PMCID: PMC4972246.
      View in: PubMed   Mentions: 7  
    114. Hip kinetics during gait are clinically meaningful outcomes in young boys with Duchenne muscular dystrophy. Gait Posture. 2016 07; 48:159-164. Heberer K, Fowler E, Staudt L, Sienko S, Buckon CE, Bagley A, Sison-Williamson M, McDonald CM, Sussman MD. PMID: 27267770.
      View in: PubMed   Mentions: 7     Fields:    Translation:Humans
    115. Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy. Neuromuscul Disord. 2016 08; 26(8):473-80. McDonald CM, Meier T, Voit T, Schara U, Straathof CS, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, Rummey C, Leinonen M, Spagnolo P, Buyse GM, DELOS Study Group. PMID: 27238057.
      View in: PubMed   Mentions: 25     Fields:    Translation:HumansCTClinical Trials
    116. Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up. Muscle Nerve. 2016 10; 54(4):681-9. Connolly AM, Florence JM, Zaidman CM, Golumbek PT, Mendell JR, Flanigan KM, Karachunski PI, Day JW, McDonald CM, Darras BT, Kang PB, Siener CA, Gadeken RK, Anand P, Schierbecker JR, Malkus EC, Lowes LP, Alfano LN, Johnson L, Nicorici A, Kelecic JM, Quigley J, Pasternak AE, Miller JP, MDA-DMD Clinical Research Network. PMID: 26930423.
      View in: PubMed   Mentions: 13     Fields:    Translation:Humans
    117. Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study. PLoS One. 2016; 11(4):e0153461. Boca SM, Nishida M, Harris M, Rao S, Cheema AK, Gill K, Seol H, Morgenroth LP, Henricson E, McDonald C, Mah JK, Clemens PR, Hoffman EP, Hathout Y, Madhavan S. PMID: 27082433; PMCID: PMC4833348.
      View in: PubMed   Mentions: 17     Fields:    Translation:Humans
    118. Is high-dose β-lactam therapy associated with excessive drug toxicity in critically ill patients? Minerva Anestesiol. 2016 Sep; 82(9):957-65. McDonald C, Cotta MO, Little PJ, McWhinney B, Ungerer JP, Lipman J, Roberts JA. PMID: 27054905.
      View in: PubMed   Mentions: 21     Fields:    Translation:Humans
    119. Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study. Ann Clin Transl Neurol. 2016 02; 3(2):132-45. Kolb SJ, Coffey CS, Yankey JW, Krosschell K, Arnold WD, Rutkove SB, Swoboda KJ, Reyna SP, Sakonju A, Darras BT, Shell R, Kuntz N, Castro D, Iannaccone ST, Parsons J, Connolly AM, Chiriboga CA, McDonald C, Burnette WB, Werner K, Thangarajh M, Shieh PB, Finanger E, Cudkowicz ME, McGovern MM, McNeil DE, Finkel R, Kaye E, Kingsley A, Renusch SR, McGovern VL, Wang X, Zaworski PG, Prior TW, Burghes AH, Bartlett A, Kissel JT, NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators. PMID: 26900585; PMCID: PMC4748311.
      View in: PubMed   Mentions: 59     Fields:    
    120. ICU-Acquired Weakness Is Associated With Differences in Clinical Outcomes in Critically Ill Children. Pediatr Crit Care Med. 2016 Jan; 17(1):53-7. Field-Ridley A, Dharmar M, Steinhorn D, McDonald C, Marcin JP. PMID: 26492063; PMCID: PMC5008971.
      View in: PubMed   Mentions: 26     Fields:    Translation:Humans
    121. Reachable workspace and performance of upper limb (PUL) in duchenne muscular dystrophy. Muscle Nerve. 2016 Apr; 53(4):545-54. Han JJ, de Bie E, Nicorici A, Abresch RT, Anthonisen C, Bajcsy R, Kurillo G, Mcdonald CM. PMID: 26342193; PMCID: PMC4779432.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    122. Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study. Neurology. 2015 Sep 22; 85(12):1048-55. Bello L, Gordish-Dressman H, Morgenroth LP, Henricson EK, Duong T, Hoffman EP, Cnaan A, McDonald CM, CINRG Investigators. PMID: 26311750; PMCID: PMC4603595.
      View in: PubMed   Mentions: 87     Fields:    Translation:Humans
    123. How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration. Orphanet J Rare Dis. 2015 Jun 24; 10:82. Furlong P, Bridges JF, Charnas L, Fallon JR, Fischer R, Flanigan KM, Franson TR, Gulati N, McDonald C, Peay H, Sweeney HL. PMID: 26104810; PMCID: PMC4486430.
      View in: PubMed   Mentions: 22     Fields:    
    124. Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy. Proc Natl Acad Sci U S A. 2015 Jun 09; 112(23):7153-8. Hathout Y, Brody E, Clemens PR, Cripe L, DeLisle RK, Furlong P, Gordish-Dressman H, Hache L, Henricson E, Hoffman EP, Kobayashi YM, Lorts A, Mah JK, McDonald C, Mehler B, Nelson S, Nikrad M, Singer B, Steele F, Sterling D, Sweeney HL, Williams S, Gold L. PMID: 26039989; PMCID: PMC4466703.
      View in: PubMed   Mentions: 148     Fields:    Translation:Humans
    125. Feasibility and Reproducibility of Echocardiographic Measures in Children with Muscular Dystrophies. J Am Soc Echocardiogr. 2015 Aug; 28(8):999-1008. Spurney CF, McCaffrey FM, Cnaan A, Morgenroth LP, Ghelani SJ, Gordish-Dressman H, Arrieta A, Connolly AM, Lotze TE, McDonald CM, Leshner RT, Clemens PR. PMID: 25906753; PMCID: PMC4526320.
      View in: PubMed   Mentions: 26     Fields:    Translation:Humans
    126. Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial. Lancet. 2015 May 02; 385(9979):1748-1757. Buyse GM, Voit T, Schara U, Straathof CSM, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, McDonald CM, Rummey C, Meier T, DELOS Study Group. PMID: 25907158.
      View in: PubMed   Mentions: 60     Fields:    Translation:HumansCTClinical Trials
    127. Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study. Ann Neurol. 2015 Apr; 77(4):684-96. Bello L, Kesari A, Gordish-Dressman H, Cnaan A, Morgenroth LP, Punetha J, Duong T, Henricson EK, Pegoraro E, McDonald CM, Hoffman EP, Cooperative International Neuromuscular Research Group Investigators. PMID: 25641372; PMCID: PMC4403971.
      View in: PubMed   Mentions: 74     Fields:    Translation:Humans
    128. Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45. Ann Neurol. 2015 Apr; 77(4):668-74. Findlay AR, Wein N, Kaminoh Y, Taylor LE, Dunn DM, Mendell JR, King WM, Pestronk A, Florence JM, Mathews KD, Finkel RS, Swoboda KJ, Howard MT, Day JW, McDonald C, Nicolas A, Le Rumeur E, Weiss RB, Flanigan KM, United Dystrophinopathy Project. PMID: 25612243; PMCID: PMC4376581.
      View in: PubMed   Mentions: 25     Fields:    Translation:Humans
    129. Outcome reliability in non-ambulatory boys/men with Duchenne muscular dystrophy. Muscle Nerve. 2015 Apr; 51(4):522-32. Connolly AM, Malkus EC, Mendell JR, Flanigan KM, Miller JP, Schierbecker JR, Siener CA, Golumbek PT, Zaidman CM, Mcdonald CM, Johnson L, Nicorici A, Karachunski PI, Day JW, Kelecic JM, Lowes LP, Alfano LN, Darras BT, Kang PB, Quigley J, Pasternak AE, Florence JM, MDA DMD Clinical Research Network. PMID: 25056178; PMCID: PMC4305351.
      View in: PubMed   Mentions: 28     Fields:    Translation:Humans
    130. Evaluation of phrenic nerve and diaphragm function with peripheral nerve stimulation and M-mode ultrasonography in potential pediatric phrenic nerve or diaphragm pacing candidates. Phys Med Rehabil Clin N Am. 2015 Feb; 26(1):133-43. Skalsky AJ, Lesser DJ, McDonald CM. PMID: 25479785.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    131. Predicting Age at Loss of Ambulation in Duchenne Muscular Dystrophy with Deep Phenotypic Measures. . 2014 Dec 1; 1344-1347. Wang WY, Bello BL, Wang WY, McDonald MC, Hoffman HE, Yu YG. .
      View in: Publisher Site   Mentions:
    132. Ataluren treatment of patients with nonsense mutation dystrophinopathy. Muscle Nerve. 2014 Oct; 50(4):477-87. Bushby K, Finkel R, Wong B, Barohn R, Campbell C, Comi GP, Connolly AM, Day JW, Flanigan KM, Goemans N, Jones KJ, Mercuri E, Quinlivan R, Renfroe JB, Russman B, Ryan MM, Tulinius M, Voit T, Moore SA, Lee Sweeney H, Abresch RT, Coleman KL, Eagle M, Florence J, Gappmaier E, Glanzman AM, Henricson E, Barth J, Elfring GL, Reha A, Spiegel RJ, O'donnell MW, Peltz SW, Mcdonald CM, PTC124-GD-007-DMD STUDY GROUP. PMID: 25042182; PMCID: PMC4241581.
      View in: PubMed   Mentions: 177     Fields:    Translation:Humans
    133. Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophy. Neuromuscul Disord. 2015 Jan; 25(1):96-105. Lynn S, Aartsma-Rus A, Bushby K, Furlong P, Goemans N, De Luca A, Mayhew A, McDonald C, Mercuri E, Muntoni F, Pohlschmidt M, Verschuuren J, Voit T, Vroom E, Wells DJ, Straub V. PMID: 25307856.
      View in: PubMed   Mentions: 19     Fields:    Translation:HumansAnimals
    134. Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patients. Hum Mol Genet. 2014 Dec 15; 23(24):6458-69. Hathout Y, Marathi RL, Rayavarapu S, Zhang A, Brown KJ, Seol H, Gordish-Dressman H, Cirak S, Bello L, Nagaraju K, Partridge T, Hoffman EP, Takeda S, Mah JK, Henricson E, McDonald C. PMID: 25027324; PMCID: PMC4240201.
      View in: PubMed   Mentions: 68     Fields:    Translation:HumansAnimals
    135. One year outcome of boys with Duchenne muscular dystrophy using the Bayley-III scales of infant and toddler development. Pediatr Neurol. 2014 Jun; 50(6):557-63. Connolly AM, Florence JM, Cradock MM, Eagle M, Flanigan KM, McDonald CM, Karachunski PI, Darras BT, Bushby K, Malkus EC, Golumbek PT, Zaidman CM, Miller JP, Mendell JR, MDA DMD Clinical Research Network. PMID: 24842254; PMCID: PMC4197452.
      View in: PubMed   Mentions: 17     Fields:    Translation:Humans
    136. A randomized, double-blind trial of lisinopril and losartan for the treatment of cardiomyopathy in duchenne muscular dystrophy. PLoS Curr. 2013 Dec 12; 5. Allen HD, Flanigan KM, Thrush PT, Dvorchik I, Yin H, Canter C, Connolly AM, Parrish M, McDonald CM, Braunlin E, Colan SD, Day J, Darras B, Mendell JR. PMID: 24459612; PMCID: PMC3871420.
      View in: PubMed   Mentions: 33  
    137. Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy. Dev Med Child Neurol. 2013 Nov; 55(11):1038-45. Mayhew A, Mazzone ES, Eagle M, Duong T, Ash M, Decostre V, Vandenhauwe M, Klingels K, Florence J, Main M, Bianco F, Henrikson E, Servais L, Campion G, Vroom E, Ricotti V, Goemans N, McDonald C, Mercuri E, Performance of the Upper Limb Working Group. PMID: 23902233.
      View in: PubMed   Mentions: 81     Fields:    Translation:Humans
    138. Why short stature is beneficial in Duchenne muscular dystrophy. Muscle Nerve. 2013 Sep; 48(3):336-42. Bodor M, McDonald CM. PMID: 23893308.
      View in: PubMed   Mentions: 16     Fields:    Translation:HumansAnimalsCells
    139. The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study. Muscle Nerve. 2013 Sep; 48(3):357-68. McDonald CM, Henricson EK, Abresch RT, Florence J, Eagle M, Gappmaier E, Glanzman AM, PTC124-GD-007-DMD Study Group, Spiegel R, Barth J, Elfring G, Reha A, Peltz SW. PMID: 23674289; PMCID: PMC3826053.
      View in: PubMed   Mentions: 119     Fields:    Translation:Humans
    140. The 6-Minute Walk Test and Person-Reported Outcomes in Boys with Duchenne Muscular Dystrophy and Typically Developing Controls: Longitudinal Comparisons and Clinically-Meaningful Changes Over One Year. PLoS Curr. 2013 Jul 08; 5. Henricson E, Abresch R, Han JJ, Nicorici A, Goude Keller E, de Bie E, McDonald CM. PMID: 23867975.
      View in: PubMed   Mentions: 52  
    141. The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study. Muscle Nerve. 2013 Sep; 48(3):343-56. McDonald CM, Henricson EK, Abresch RT, Florence JM, Eagle M, Gappmaier E, Glanzman AM, PTC124-GD-007-DMD Study Group, Spiegel R, Barth J, Elfring G, Reha A, Peltz S. PMID: 23681930; PMCID: PMC3824082.
      View in: PubMed   Mentions: 134     Fields:    Translation:Humans
    142. Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network. Neuromuscul Disord. 2013 Jul; 23(7):529-39. Connolly AM, Florence JM, Cradock MM, Malkus EC, Schierbecker JR, Siener CA, Wulf CO, Anand P, Golumbek PT, Zaidman CM, Philip Miller J, Lowes LP, Alfano LN, Viollet-Callendret L, Flanigan KM, Mendell JR, McDonald CM, Goude E, Johnson L, Nicorici A, Karachunski PI, Day JW, Dalton JC, Farber JM, Buser KK, Darras BT, Kang PB, Riley SO, Shriber E, Parad R, Bushby K, Eagle M, MDA DMD Clinical Research Network. PMID: 23726376; PMCID: PMC3743677.
      View in: PubMed   Mentions: 34     Fields:    Translation:Humans
    143. The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used. Muscle Nerve. 2013 Jul; 48(1):32-54. McDonald CM, Henricson EK, Abresch RT, Han JJ, Escolar DM, Florence JM, Duong T, Arrieta A, Clemens PR, Hoffman EP, Cnaan A, Cinrg Investigators. PMID: 23677550; PMCID: PMC4147958.
      View in: PubMed   Mentions: 86     Fields:    Translation:Humans
    144. The cooperative international neuromuscular research group Duchenne natural history study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures. Muscle Nerve. 2013 Jul; 48(1):55-67. Henricson EK, Abresch RT, Cnaan A, Hu F, Duong T, Arrieta A, Han J, Escolar DM, Florence JM, Clemens PR, Hoffman EP, McDonald CM, CINRG Investigators. PMID: 23649481; PMCID: PMC4103170.
      View in: PubMed   Mentions: 93     Fields:    Translation:Humans
    145. LTBP4 genotype predicts age of ambulatory loss in Duchenne muscular dystrophy. Ann Neurol. 2013 Apr; 73(4):481-8. Flanigan KM, Ceco E, Lamar KM, Kaminoh Y, Dunn DM, Mendell JR, King WM, Pestronk A, Florence JM, Mathews KD, Finkel RS, Swoboda KJ, Gappmaier E, Howard MT, Day JW, McDonald C, McNally EM, Weiss RB, United Dystrophinopathy Project. PMID: 23440719; PMCID: PMC4106425.
      View in: PubMed   Mentions: 133     Fields:    Translation:HumansCells
    146. Health and fitness in pediatric spinal cord injury: medical issues and the role of exercise. J Pediatr Rehabil Med. 2013; 6(1):35-44. Johnston TE, McDonald CM. PMID: 23481890.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    147. Management of pulmonary complications in neuromuscular disease. Phys Med Rehabil Clin N Am. 2012 Nov; 23(4):829-53. Wolfe LF, Joyce NC, McDonald CM, Benditt JO, Finder J. PMID: 23137740.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    148. Treatment of spine deformity in neuromuscular diseases. Phys Med Rehabil Clin N Am. 2012 Nov; 23(4):869-83. Maitra S, Roberto RF, McDonald CM, Gupta MC. PMID: 23137742.
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    149. Mobility-assistive technology in progressive neuromuscular disease. Phys Med Rehabil Clin N Am. 2012 Nov; 23(4):885-94. Lin W, Pierce A, Skalsky AJ, McDonald CM. PMID: 23137743.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    150. Neuromuscular disease management and rehabilitation, part II: specialty care and therapeutics. Phys Med Rehabil Clin N Am. 2012 Nov; 23(4):xiii-xvii. McDonald CM, Joyce NC. PMID: 23137747.
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    151. Exercise in neuromuscular diseases. Phys Med Rehabil Clin N Am. 2012 Aug; 23(3):653-73. Abresch RT, Carter GT, Han JJ, McDonald CM. PMID: 22938880.
      View in: PubMed   Mentions: 20     Fields:    Translation:Humans
    152. Neuromuscular disease management and rehabilitation, part I: diagnostic and therapy issues. Phys Med Rehabil Clin N Am. 2012 Aug; 23(3):xvii-xx. McDonald CM, Joyce NC. PMID: 22938886.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    153. Clinical approach to the diagnostic evaluation of hereditary and acquired neuromuscular diseases. Phys Med Rehabil Clin N Am. 2012 Aug; 23(3):495-563. McDonald CM. PMID: 22938875; PMCID: PMC3482409.
      View in: PubMed   Mentions: 31     Fields:    Translation:Humans
    154. William M. Fowler, Jr., MD. Dedication. Phys Med Rehabil Clin N Am. 2012 Aug; 23(3):xxi-xxii. McDonald CM. PMID: 22938887.
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    155. The role of the neuromuscular medicine and physiatry specialists in the multidisciplinary management of neuromuscular disease. Phys Med Rehabil Clin N Am. 2012 Aug; 23(3):475-93. McDonald CM, Fowler WM. PMID: 22938874; PMCID: PMC3482408.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    156. Prevention and management of limb contractures in neuromuscular diseases. Phys Med Rehabil Clin N Am. 2012 Aug; 23(3):675-87. Skalsky AJ, McDonald CM. PMID: 22938881; PMCID: PMC3482407.
      View in: PubMed   Mentions: 44     Fields:    Translation:Humans
    157. International workshop on assessment of upper limb function in Duchenne Muscular Dystrophy: Rome, 15-16 February 2012. Neuromuscul Disord. 2012 Nov; 22(11):1025-8. Mercuri E, McDonald C, Mayhew A, Florence J, Mazzone E, Bianco F, Decostre V, Servais L, Ricotti V, Goemans N, Vroom E. PMID: 22795657; PMCID: PMC3500683.
      View in: PubMed   Mentions: 9     Fields:    Translation:Humans
    158. Corticosteroids and Duchenne muscular dystrophy: does earlier treatment really matter? Muscle Nerve. 2012 Jun; 45(6):777-9. McDonald CM, Han JJ, Mah JK, Carter GT. PMID: 22581529.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    159. Percent-predicted 6-minute walk distance in duchenne muscular dystrophy to account for maturational influences. PLoS Curr. 2012 Jan 25; 3:RRN1297. Henricson E, Abresch R, Han JJ, Nicorici A, Goude Keller E, Elfring G, Reha A, Barth J, McDonald CM. PMID: 22306689; PMCID: PMC3269886.1.
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    160. Regional and whole-body dual-energy X-ray absorptiometry to guide treatment and monitor disease progression in neuromuscular disease. Phys Med Rehabil Clin N Am. 2012 Feb; 23(1):67-73, x. Skalsky AJ, Han JJ, Abresch RT, McDonald CM. PMID: 22239875.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    161. Quality-of-life measures in children with neurological conditions: pediatric Neuro-QOL. Neurorehabil Neural Repair. 2012 Jan; 26(1):36-47. Lai JS, Nowinski C, Victorson D, Bode R, Podrabsky T, McKinney N, Straube D, Holmes GL, McDonald CM, Henricson E, Abresch RT, Moy CS, Cella D. PMID: 21788436; PMCID: PMC3710728.
      View in: PubMed   Mentions: 37     Fields:    Translation:Humans
    162. Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. Neurology. 2011 Aug 02; 77(5):444-52. Escolar DM, Hache LP, Clemens PR, Cnaan A, McDonald CM, Viswanathan V, Kornberg AJ, Bertorini TE, Nevo Y, Lotze T, Pestronk A, Ryan MM, Monasterio E, Day JW, Zimmerman A, Arrieta A, Henricson E, Mayhew J, Florence J, Hu F, Connolly AM. PMID: 21753160; PMCID: PMC3146308.
      View in: PubMed   Mentions: 68     Fields:    Translation:Humans
    163. The natural history of cardiac and pulmonary function decline in patients with duchenne muscular dystrophy. Spine (Phila Pa 1976). 2011 Jul 01; 36(15):E1009-17. Roberto R, Fritz A, Hagar Y, Boice B, Skalsky A, Hwang H, Beckett L, McDonald C, Gupta M. PMID: 21289561; PMCID: PMC3120906.
      View in: PubMed   Mentions: 20     Fields:    Translation:Humans
    164. Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene. Hum Mutat. 2011 Mar; 32(3):299-308. Flanigan KM, Dunn DM, von Niederhausern A, Soltanzadeh P, Howard MT, Sampson JB, Swoboda KJ, Bromberg MB, Mendell JR, Taylor LE, Anderson CB, Pestronk A, Florence JM, Connolly AM, Mathews KD, Wong B, Finkel RS, Bonnemann CG, Day JW, McDonald C, United Dystrophinopathy Project Consortium, Weiss RB. PMID: 21972111; PMCID: PMC3724403.
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    165. SPP1 genotype is a determinant of disease severity in Duchenne muscular dystrophy. Neurology. 2011 Jan 18; 76(3):219-26. Pegoraro E, Hoffman EP, Piva L, Gavassini BF, Cagnin S, Ermani M, Bello L, Soraru G, Pacchioni B, Bonifati MD, Lanfranchi G, Angelini C, Kesari A, Lee I, Gordish-Dressman H, Devaney JM, McDonald CM, Cooperative International Neuromuscular Research Group. PMID: 21178099; PMCID: PMC3034396.
      View in: PubMed   Mentions: 120     Fields:    Translation:Humans
    166. The 6-minute walk test in Duchenne/Becker muscular dystrophy: longitudinal observations. Muscle Nerve. 2010 Dec; 42(6):966-74. McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Atkinson L, Elfring GL, Reha A, Miller LL. PMID: 21038378.
      View in: PubMed   Mentions: 63     Fields:    Translation:Humans
    167. Response to Letter to the Editor: New Clinical Endpoints in Rehabilitation Medicine. American Journal of Hospice and Palliative Medicine®. 2010 Aug 1; 27(5):359-359. Abresch AR, Carter CG, Han HJ, McDonald MC. .
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    168. Classification of the gait patterns of boys with Duchenne muscular dystrophy and their relationship to function. J Child Neurol. 2010 Sep; 25(9):1103-9. Sienko Thomas S, Buckon CE, Nicorici A, Bagley A, McDonald CM, Sussman MD. PMID: 20587736; PMCID: PMC3794706.
      View in: PubMed   Mentions: 22     Fields:    Translation:Humans
    169. Relationship between clinical outcome measures and parent proxy reports of health-related quality of life in ambulatory children with Duchenne muscular dystrophy. J Child Neurol. 2010 Sep; 25(9):1130-44. McDonald CM, McDonald DA, Bagley A, Sienko Thomas S, Buckon CE, Henricson E, Nicorici A, Sussman MD. PMID: 20558672; PMCID: PMC3695468.
      View in: PubMed   Mentions: 24     Fields:    Translation:Humans
    170. The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy. Muscle Nerve. 2010 Apr; 41(4):500-10. McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Elfring GL, Atkinson L, Reha A, Hirawat S, Miller LL. PMID: 19941337.
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    171. New clinical end points in rehabilitation medicine: tools for measuring quality of life. Am J Hosp Palliat Care. 2009 Dec-2010 Jan; 26(6):483-92. Abresch RT, Carter GT, Han JJ, McDonald CM. PMID: 19923631.
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    172. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol. 2010 Feb; 9(2):177-89. Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C, DMD Care Considerations Working Group. PMID: 19945914.
      View in: PubMed   Mentions: 358     Fields:    Translation:Humans
    173. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010 Jan; 9(1):77-93. Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C, DMD Care Considerations Working Group. PMID: 19945913.
      View in: PubMed   Mentions: 662     Fields:    Translation:Humans
    174. Development of items designed to evaluate activity performance and participation in children and adolescents with spinal cord injury. Int J Pediatr. 2009; 2009:854904. Calhoun CL, Haley SM, Riley A, Vogel LC, McDonald CM, Mulcahey MJ. PMID: 20049343; PMCID: PMC2798099.
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    175. Impact of biopsychosocial factors on chronic pain in persons with myotonic and facioscapulohumeral muscular dystrophy. Am J Hosp Palliat Care. 2009 Aug-Sep; 26(4):308-19. Miró J, Raichle KA, Carter GT, O'Brien SA, Abresch RT, McDonald CM, Jensen MP. PMID: 19414560; PMCID: PMC2845314.
      View in: PubMed   Mentions: 18     Fields:    Translation:Humans
    176. Assessment of regional body composition with dual-energy X-ray absorptiometry in Duchenne muscular dystrophy: correlation of regional lean mass and quantitative strength. Muscle Nerve. 2009 May; 39(5):647-51. Skalsky AJ, Han JJ, Abresch RT, Shin CS, McDonald CM. PMID: 19347922.
      View in: PubMed   Mentions: 23     Fields:    Translation:Humans
    177. Change in life satisfaction of adults with pediatric-onset spinal cord injury. Arch Phys Med Rehabil. 2008 Dec; 89(12):2285-92. Chen Y, Anderson CJ, Vogel LC, Chlan KM, Betz RR, McDonald CM. PMID: 19061740.
      View in: PubMed   Mentions: 9     Fields:    Translation:Humans
    178. Pain in myotonic muscular dystrophy, type 1. Arch Phys Med Rehabil. 2008 Dec; 89(12):2382. Carter GT, Jensen MP, Hoffman AJ, Stoelb BL, Abresch RT, McDonald CM. PMID: 19061752.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    179. Pain in persons with postpolio syndrome: frequency, intensity, and impact. Arch Phys Med Rehabil. 2008 Oct; 89(10):1933-40. Stoelb BL, Carter GT, Abresch RT, Purekal S, McDonald CM, Jensen MP. PMID: 18929021; PMCID: PMC2651567.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    180. Convexity of scoliosis related to handedness in identical twin boys with Duchenne's muscular dystrophy: a case report. Arch Phys Med Rehabil. 2008 Oct; 89(10):2021-4. Werner BC, Skalsky AJ, McDonald CM, Han JJ. PMID: 18929032.
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    181. The relationship between regional body composition and quantitative strength in facioscapulohumeral muscular dystrophy (FSHD). Neuromuscul Disord. 2008 Nov; 18(11):873-80. Skalsky AJ, Abresch RT, Han JJ, Shin CS, McDonald CM. PMID: 18818077.
      View in: PubMed   Mentions: 9     Fields:    Translation:Humans
    182. Motor scores on the functional independence measure after pediatric spinal cord injury. Spinal Cord. 2009 Mar; 47(3):213-7. Allen DD, Mulcahey MJ, Haley SM, Devivo MJ, Vogel LC, McDonald C, Duffy T, Betz RR. PMID: 18679405; PMCID: PMC2718680.
      View in: PubMed   Mentions: 9     Fields:    Translation:Humans
    183. Diagnosis and clinical management of spinal muscular atrophy. Phys Med Rehabil Clin N Am. 2008 Aug; 19(3):661-80, xii. Han JJ, McDonald CM. PMID: 18625423.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    184. The validity of compliance monitors to assess wearing time of thoracic-lumbar-sacral orthoses in children with spinal cord injury. Spine (Phila Pa 1976). 2008 Jun 15; 33(14):1554-61. Hunter LN, Sison-Williamson M, Mendoza MM, McDonald CM, Molitor F, Mulcahey MJ, Betz RR, Vogel LC, Bagley A. PMID: 18552670.
      View in: PubMed   Mentions: 8     Fields:    Translation:Humans
    185. Variable phenotypes associated with mutations in DOK7. Muscle Nerve. 2008 Apr; 37(4):448-56. Anderson JA, Ng JJ, Bowe C, McDonald C, Richman DP, Wollmann RL, Maselli RA. PMID: 18161030.
      View in: PubMed   Mentions: 17     Fields:    Translation:Humans
    186. Chronic pain in persons with myotonic dystrophy and facioscapulohumeral dystrophy. Arch Phys Med Rehabil. 2008 Feb; 89(2):320-8. Jensen MP, Hoffman AJ, Stoelb BL, Abresch RT, Carter GT, McDonald CM. PMID: 18226657; PMCID: PMC2828945.
      View in: PubMed   Mentions: 33     Fields:    Translation:Humans
    187. Reliability of radiographic parameters in neuromuscular scoliosis. Spine (Phila Pa 1976). 2007 Mar 15; 32(6):691-5. Gupta MC, Wijesekera S, Sossan A, Martin L, Vogel LC, Boakes JL, Lerman JA, McDonald CM, Betz RR. PMID: 17413476.
      View in: PubMed   Mentions: 13     Fields:    Translation:Humans
    188. Depression in adults who sustained spinal cord injuries as children or adolescents. J Spinal Cord Med. 2007; 30 Suppl 1:S76-82. Anderson CJ, Vogel LC, Chlan KM, Betz RR, McDonald CM. PMID: 17874691; PMCID: PMC2031993.
      View in: PubMed   Mentions: 23     Fields:    Translation:Humans
    189. Challenges in drug development for muscle disease: a stakeholders' meeting. Muscle Nerve. 2007 Jan; 35(1):8-16. Mendell JR, Csimma C, McDonald CM, Escolar DM, Janis S, Porter JD, Hesterlee SE, Howell RR. PMID: 17068768.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansAnimals
    190. Impact of prophylactic thoracolumbosacral orthosis bracing on functional activities and activities of daily living in the pediatric spinal cord injury population. J Spinal Cord Med. 2007; 30 Suppl 1:S178-83. Chafetz RS, Mulcahey MJ, Betz RR, Anderson C, Vogel LC, Gaughan JP, Odel MA, Flanagan A, McDonald CM. PMID: 17874705; PMCID: PMC2031986.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    191. Aerobic fitness and upper extremity strength in patients aged 11 to 21 years with spinal cord dysfunction as compared to ideal weight and overweight controls. J Spinal Cord Med. 2007; 30 Suppl 1:S88-96. Widman LM, Abresch RT, Styne DM, McDonald CM. PMID: 17874693; PMCID: PMC2031980.
      View in: PubMed   Mentions: 18     Fields:    Translation:Humans
    192. Body mass index and body composition measures by dual x-ray absorptiometry in patients aged 10 to 21 years with spinal cord injury. J Spinal Cord Med. 2007; 30 Suppl 1:S97-104. McDonald CM, Abresch-Meyer AL, Nelson MD, Widman LM. PMID: 17874694; PMCID: PMC2031982.
      View in: PubMed   Mentions: 29     Fields:    Translation:Humans
    193. Development and pilot test of the shriners pediatric instrument for neuromuscular scoliosis (SPNS): a quality of life questionnaire for children with spinal cord injuries. J Spinal Cord Med. 2007; 30 Suppl 1:S150-7. Hunter L, Molitor F, Chafetz RS, Mulcahey MJ, Vogel LC, Betz RR, McDonald CM. PMID: 17874701; PMCID: PMC2031972.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    194. Effect of thoracolumbosacral orthoses on reachable workspace volumes in children with spinal cord injury. J Spinal Cord Med. 2007; 30 Suppl 1:S184-91. Sison-Williamson M, Bagley A, Hongo A, Vogel LC, Mulcahey MJ, Betz RR, McDonald CM. PMID: 17874706; PMCID: PMC2031977.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansCTClinical Trials
    195. Behavioral intervention, exercise, and nutrition education to improve health and fitness (BENEfit) in adolescents with mobility impairment due to spinal cord dysfunction. J Spinal Cord Med. 2007; 30 Suppl 1:S119-26. Liusuwan RA, Widman LM, Abresch RT, Johnson AJ, McDonald CM. PMID: 17874697; PMCID: PMC2031996.
      View in: PubMed   Mentions: 11     Fields:    Translation:HumansCTClinical Trials
    196. Body composition and resting energy expenditure in patients aged 11 to 21 years with spinal cord dysfunction compared to controls: comparisons and relationships among the groups. J Spinal Cord Med. 2007; 30 Suppl 1:S105-11. Liusuwan RA, Widman LM, Abresch RT, Styne DM, McDonald CM. PMID: 17874695; PMCID: PMC2031969.
      View in: PubMed   Mentions: 25     Fields:    Translation:Humans
    197. Metabolic syndrome in adolescents with spinal cord dysfunction. J Spinal Cord Med. 2007; 30 Suppl 1:S127-39. Nelson MD, Widman LM, Abresch RT, Stanhope K, Havel PJ, Styne DM, McDonald CM. PMID: 17874698; PMCID: PMC2031992.
      View in: PubMed   Mentions: 49     Fields:    Translation:Humans
    198. A prospective evaluation of the WeeFIM in patients with cerebral palsy undergoing orthopaedic surgery. J Pediatr Orthop. 2006 Jul-Aug; 26(4):542-6. Sanders JO, McConnell SL, King R, Lanford A, Montpetit K, Gates P, Rich MM, Shepherd K, Cupp T, Haynes R, Bush P, Tahir F, Santiago J, Lighter DE, Smrcina C, Niederpruem ML, McDonald C, Curry DB. PMID: 16791077.
      View in: PubMed   Mentions: 7     Fields:    Translation:Humans
    199. Effectiveness of an upper extremity exercise device integrated with computer gaming for aerobic training in adolescents with spinal cord dysfunction. J Spinal Cord Med. 2006; 29(4):363-70. Widman LM, McDonald CM, Abresch RT. PMID: 17044386; PMCID: PMC1864855.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansCTClinical Trials
    200. Body composition and water compartment measurements in boys with Duchenne muscular dystrophy. Am J Phys Med Rehabil. 2005 Jul; 84(7):483-91. McDonald CM, Carter GT, Abresch RT, Widman L, Styne DM, Warden N, Kilmer DD. PMID: 15973084.
      View in: PubMed   Mentions: 10     Fields:    Translation:Humans
    201. Chronic pain in persons with neuromuscular disease. Arch Phys Med Rehabil. 2005 Jun; 86(6):1155-63. Jensen MP, Abresch RT, Carter GT, McDonald CM. PMID: 15954054.
      View in: PubMed   Mentions: 34     Fields:    Translation:Humans
    202. Utility of a step activity monitor for the measurement of daily ambulatory activity in children. Arch Phys Med Rehabil. 2005 Apr; 86(4):793-801. McDonald CM, Widman L, Abresch RT, Walsh SA, Walsh DD. PMID: 15827934.
      View in: PubMed   Mentions: 30     Fields:    Translation:Humans
    203. Use of step activity monitoring for continuous physical activity assessment in boys with Duchenne muscular dystrophy. Arch Phys Med Rehabil. 2005 Apr; 86(4):802-8. McDonald CM, Widman LM, Walsh DD, Walsh SA, Abresch RT. PMID: 15827935.
      View in: PubMed   Mentions: 31     Fields:    Translation:Humans
    204. Childhood neurological disorders: crosscutting breakout session. Physical Disabilities through the Lifespan Conference. Neurorehabil Neural Repair. 2005 Mar; 19(1 Suppl):90S-3S. PMID: 15689483.
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    205. Practice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology. 2005 Jan 11; 64(1):13-20. Moxley RT, Ashwal S, Pandya S, Connolly A, Florence J, Mathews K, Baumbach L, McDonald C, Sussman M, Wade C, Quality Standards Subcommittee of the American Academy of Neurology, Practice Committee of the Child Neurology Society. PMID: 15642897.
      View in: PubMed   Mentions: 85     Fields:    Translation:Humans
    206. Recognition and management of autonomic dysreflexia in pediatric spinal cord injury. J Spinal Cord Med. 2004; 27 Suppl 1:S61-74. McGinnis KB, Vogel LC, McDonald CM, Porth S, Hickey KJ, Davis M, Bush P, Jenkins D, Shriners Hospitals for Children Task Force on Autonomic Dysreflexia in Children with Spinal Cord Inj. PMID: 15503705.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    207. Altered body composition affects resting energy expenditure and interpretation of body mass index in children with spinal cord injury. J Spinal Cord Med. 2004; 27 Suppl 1:S24-8. Liusuwan A, Widman L, Abresch RT, McDonald CM. PMID: 15503699.
      View in: PubMed   Mentions: 19     Fields:    Translation:Humans
    208. Leisure characteristics of the pediatric spinal cord injury population. J Spinal Cord Med. 2004; 27 Suppl 1:S107-9. Johnson KA, Klaas SJ, Vogel LC, McDonald C. PMID: 15503712.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    209. Timed motor test for wheelchair users: initial development and application in children with spinal cord injury. J Spinal Cord Med. 2004; 27 Suppl 1:S38-43. Chafetz R, McDonald C, Mulcahey MJ, Betz R, Anderson C, Vogel L, Gaughan JP, Martin S, O'Dell MA, Flanagan A. PMID: 15503701.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    210. Effect of bracing on paralytic scoliosis secondary to spinal cord injury. J Spinal Cord Med. 2004; 27 Suppl 1:S88-92. Mehta S, Betz RR, Mulcahey MJ, McDonald C, Vogel LC, Anderson C. PMID: 15503709.
      View in: PubMed   Mentions: 18     Fields:    Translation:Humans
    211. Physical activity, health impairments, and disability in neuromuscular disease. Am J Phys Med Rehabil. 2002 Nov; 81(11 Suppl):S108-20. McDonald CM. PMID: 12409816.
      View in: PubMed   Mentions: 47     Fields:    Translation:Humans
    212. Presynaptic congenital myasthenic syndrome due to quantal release deficiency. Neurology. 2001 Jul 24; 57(2):279-89. Maselli RA, Kong DZ, Bowe CM, McDonald CM, Ellis WG, Agius MA, Gomez CM, Richman DP, Wollmann RL. PMID: 11468313.
      View in: PubMed   Mentions: 5     Fields:    Translation:HumansCells
    213. Ultrasonographic evaluation of diaphragmatic motion. J Ultrasound Med. 2001 Jun; 20(6):597-604. Gerscovich EO, Cronan M, McGahan JP, Jain K, Jones CD, McDonald C. PMID: 11400933.
      View in: PubMed   Mentions: 67     Fields:    Translation:Humans
    214. Peripheral neuropathies of childhood. Phys Med Rehabil Clin N Am. 2001 May; 12(2):473-90. McDonald CM. PMID: 11345019.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    215. Magnetic resonance imaging of denervated muscle: comparison to electromyography. Muscle Nerve. 2000 Sep; 23(9):1431-4. McDonald CM, Carter GT, Fritz RC, Anderson MW, Abresch RT, Kilmer DD. PMID: 10951448.
      View in: PubMed   Mentions: 21     Fields:    Translation:Humans
    216. Preserving function in Duchenne dystrophy with long-term pulse prednisone therapy. Am J Phys Med Rehabil. 2000 Sep-Oct; 79(5):455-8. Carter GT, McDonald CM. PMID: 10994887.
      View in: PubMed   Mentions: 9     Fields:    Translation:Humans
    217. Spinal sonography and magnetic resonance imaging in patients with repaired myelomeningocele: comparison of modalities. J Ultrasound Med. 1999 Sep; 18(9):655-64. Gerscovich EO, Maslen L, Cronan MS, Poirier V, Anderson MW, McDonald C, Boggan JE, Ivanovic M. PMID: 10478975.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    218. Mutations in the early growth response 2 (EGR2) gene are associated with hereditary myelinopathies. Nat Genet. 1998 Apr; 18(4):382-4. Warner LE, Mancias P, Butler IJ, McDonald CM, Keppen L, Koob KG, Lupski JR. PMID: 9537424.
      View in: PubMed   Mentions: 122     Fields:    Translation:HumansCells
    219. Clinical approach to the diagnostic evaluation of progressive neuromuscular diseases. Phys Med Rehabil Clin N Am. 1998 Feb; 9(1):9-48. McDonald CM. PMID: 9894133.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    220. Limb contractures in progressive neuromuscular disease and the role of stretching, orthotics, and surgery. Phys Med Rehabil Clin N Am. 1998 Feb; 9(1):187-211. McDonald CM. PMID: 9894140.
      View in: PubMed   Mentions: 19     Fields:    Translation:Humans
    221. Spinal deformity in progressive neuromuscular disease. Natural history and management. Phys Med Rehabil Clin N Am. 1998 Feb; 9(1):213-32, viii. Hart DA, McDonald CM. PMID: 9894141.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    222. Fulminant demyelinating neuropathy mimicking cerebral death. Muscle Nerve. 1997 Dec; 20(12):1595-7. Bakshi N, Maselli RA, Gospe SM, Ellis WG, McDonald C, Mandler RN. PMID: 9390675.
      View in: PubMed   Mentions: 9     Fields:    Translation:Humans
    223. Profiles of functional recovery in fifty traumatically brain-injured patients after acute rehabilitation. Am J Phys Med Rehabil. 1997 May-Jun; 76(3):213-8. Davis CH, Fardanesh L, Rubner D, Wanlass RL, McDonald CM. PMID: 9207707.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    224. New approaches to managing spasticity in children with cerebral palsy. West J Med. 1997 Apr; 166(4):271. Im D, McDonald CM. PMID: 9168686; PMCID: PMC1304209.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    225. Urodynamic findings in the tethered spinal cord syndrome: does surgical release improve bladder function? J Urol. 1997 Feb; 157(2):604-9. Fone PD, Vapnek JM, Litwiller SE, Couillard DR, McDonald CM, Boggan JE, Stone AR. PMID: 8996368.
      View in: PubMed   Mentions: 12     Fields:    Translation:Humans
    226. Focal posterior interosseous neuropathy in the presence of hereditary motor and sensory neuropathy, type I. Muscle Nerve. 1996 May; 19(5):644-8. Carter GT, Kilmer DD, Szabo RM, McDonald CM. PMID: 8618563.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    227. Profiles of neuromuscular diseases. Spinal muscular atrophy. Am J Phys Med Rehabil. 1995 Sep-Oct; 74(5 Suppl):S150-9. Carter GT, Abresch RT, Fowler WM, Johnson ER, Kilmer DD, McDonald CM. PMID: 7576422.
      View in: PubMed   Mentions: 26     Fields:    Translation:Humans
    228. Profiles of neuromuscular diseases. Duchenne muscular dystrophy. Am J Phys Med Rehabil. 1995 Sep-Oct; 74(5 Suppl):S70-92. McDonald CM, Abresch RT, Carter GT, Fowler WM, Johnson ER, Kilmer DD, Sigford BJ. PMID: 7576424.
      View in: PubMed   Mentions: 119     Fields:    Translation:Humans
    229. Profiles of neuromuscular diseases. Becker's muscular dystrophy. Am J Phys Med Rehabil. 1995 Sep-Oct; 74(5 Suppl):S93-103. McDonald CM, Abresch RT, Carter GT, Fowler WM, Johnson ER, Kilmer DD. PMID: 7576425.
      View in: PubMed   Mentions: 13     Fields:    Translation:Humans
    230. Profiles of neuromuscular diseases. Limb-girdle syndromes. Am J Phys Med Rehabil. 1995 Sep-Oct; 74(5 Suppl):S117-30. McDonald CM, Johnson ER, Abresch RT, Carter GT, Fowler WM, Kilmer DD. PMID: 7576419.
      View in: PubMed   Mentions: 7     Fields:    Translation:Humans
    231. Profiles of neuromuscular diseases. Facioscapulohumeral muscular dystrophy. Am J Phys Med Rehabil. 1995 Sep-Oct; 74(5 Suppl):S131-9. Kilmer DD, Abresch RT, McCrory MA, Carter GT, Fowler WM, Johnson ER, McDonald CM. PMID: 7576420.
      View in: PubMed   Mentions: 34     Fields:    Translation:Humans
    232. Profiles of neuromuscular diseases. Hereditary motor and sensory neuropathy, types I and II. Am J Phys Med Rehabil. 1995 Sep-Oct; 74(5 Suppl):S140-9. Carter GT, Abresch RT, Fowler WM, Johnson ER, Kilmer DD, McDonald CM. PMID: 7576421.
      View in: PubMed   Mentions: 18     Fields:    Translation:Humans
    233. Rehabilitation of children with spinal dysraphism. Neurosurg Clin N Am. 1995 Apr; 6(2):393-412. McDonald CM. PMID: 7620362.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    234. Isolated femoral mononeuropathy to the vastus lateralis: EMG and MRI findings. Muscle Nerve. 1995 Mar; 18(3):341-4. Carter GT, McDonald CM, Chan TT, Margherita AJ. PMID: 7870114.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    235. Comparison of indices of traumatic brain injury severity as predictors of neurobehavioral outcome in children. Arch Phys Med Rehabil. 1994 Mar; 75(3):328-37. McDonald CM, Jaffe KM, Fay GC, Polissar NL, Martin KM, Liao S, Rivara JB. PMID: 8129588.
      View in: PubMed   Mentions: 13     Fields:    Translation:Humans
    236. Transcranial cortical stimulation in syringomyelia. Muscle Nerve. 1994 Jan; 17(1):114-6. McDonald CM, Robinson LR. PMID: 8264692.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    237. Interferon beta treatment of multiple sclerosis. Neurology. 1994 Jan; 44(1):187. McDonald MA, McDonald CM. PMID: 8290071.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    238. Distal sensory fiber involvement in inflammatory demyelinating polyneuropathies. Muscle Nerve. 1994 Jan; 17(1):112-3. Fraser JL. PMID: 8264690.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    239. Neurobehavioral and family functioning following traumatic brain injury in children. West J Med. 1992 Dec; 157(6):664. McDonald CM, Jaffe KM. PMID: 1475953; PMCID: PMC1022104.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    240. Rehabilitation following childhood injury. Pediatr Ann. 1992 Jul; 21(7):438-9, 443-7. Jaffe KM, McDonald CM. PMID: 1408415.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    241. Modifications to the traditional description of neurosegmental innervation in myelomeningocele. Dev Med Child Neurol. 1991 Jun; 33(6):473-81. McDonald CM, Jaffe KM, Shurtleff DB, Menelaus MB. PMID: 1864473.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    242. Ambulatory outcome of children with myelomeningocele: effect of lower-extremity muscle strength. Dev Med Child Neurol. 1991 Jun; 33(6):482-90. McDonald CM, Jaffe KM, Mosca VS, Shurtleff DB. PMID: 1864474.
      View in: PubMed   Mentions: 19     Fields:    Translation:Humans
    243. Symptoms of upper gastrointestinal dysfunction in Duchenne muscular dystrophy: case-control study. Arch Phys Med Rehabil. 1990 Sep; 71(10):742-4. Jaffe KM, McDonald CM, Ingman E, Haas J. PMID: 2403280.
      View in: PubMed   Mentions: 17     Fields:    Translation:Humans
    244. Assessment of muscle strength in children with meningomyelocele: accuracy and stability of measurements over time. Arch Phys Med Rehabil. 1986 Dec; 67(12):855-61. McDonald CM, Jaffe KM, Shurtleff DB. PMID: 3800612.
      View in: PubMed   Mentions: 11     Fields:    Translation:Humans
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