153 results on '"McDonald, Craig M."'
Search Results
2. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
3. Survival among patients receiving eteplirsen for up to 8 years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls
4. Gait Event Detection and Travel Distance Using Waist-Worn Accelerometers across a Range of Speeds: Automated Approach
5. Findings from the Longitudinal CINRG Becker Natural History Study
6. Contributors
7. Myopathic Disorders
8. Sleep Disordered Breathing in Children with Neuromuscular Disease
9. 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)
10. Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States
11. Safety and Efficacy of Ataluren in nmDMD Patients from Study 041, a Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial (PL5.001)
12. Ataluren Preserves Upper Limb Function in nmDMD Patients from Study 041, a Phase 3 Placebo-Controlled Trial, and the STRIDE Registry (S34.008)
13. Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status
14. Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy
15. Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial
16. Comparing Deflazacort and Prednisone in Duchenne Muscular Dystrophy
17. Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging
18. Genetic modifiers of upper limb function in Duchenne muscular dystrophy
19. Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab
20. A Longitudinal Study of Quantitative Muscle Strength and Functional Motor Ability in Ambulatory Boys with Duchenne Muscular Dystrophy
21. Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial
22. Influence of β2 adrenergic receptor genotype on longitudinal measures of forced vital capacity in patients with Duchenne muscular dystrophy
23. Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients
24. 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
25. Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy
26. Electrodiagnosis in Pediatrics
27. Neuromuscular Diseases
28. Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy
29. Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy
30. 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
31. Corrigendum to “Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy” [Neuromuscular Disorders, Vol. 30 (6) 2020, 492-502]
32. (−)‐Epicatechin induces mitochondrial biogenesis and markers of muscle regeneration in adults with Becker muscular dystrophy
33. Myopathic Disorders
34. Contributors
35. Medical management of muscle weakness in Duchenne muscular dystrophy
36. The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID‐19 pandemic
37. Conference report on contractures in musculoskeletal and neurological conditions
38. Assessment of Treatment Effect With Multiple Outcomes in 2 Clinical Trials of Patients With Duchenne Muscular Dystrophy
39. Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy
40. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study
41. Longitudinal study of upper extremity reachable workspace in fascioscapulohumeral muscular dystrophy
42. Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids
43. Home-Based Monitoring of Pulmonary Function in Patients with Duchenne Muscular Dystroph
44. Timed function tests have withstood the test of time as clinically meaningful and responsive endpoints in duchenne muscular dystrophy
45. Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug
46. Report on the workshop: Meaningful outcome measures for Duchenne muscular dystrophy, London, UK, 30–31 January 2017
47. A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial
48. Evidence-based care in Duchenne muscular dystrophy
49. Mexiletine for muscle cramps in amyotrophic lateral sclerosis: A randomized, double-blind crossover trial
50. A presynaptic congenital myasthenic syndrome attributed to a homozygous sequence variant inLAMA5
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