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356 results on '"Mah, Jean"'

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1. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

2. Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab

5. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

6. Safety and efficacy of teriflunomide in paediatric multiple sclerosis (TERIKIDS): a multicentre, double-blind, phase 3, randomised, placebo-controlled trial

7. Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy

8. Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy

9. Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study

11. Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.

12. Correction to: Impact of an electronic monitoring device and behavioural feedback on adherence to multiple sclerosis therapies in youth: results of a randomized trial

13. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study

15. Impact of an electronic monitoring device and behavioral feedback on adherence to multiple sclerosis therapies in youth: results of a randomized trial

16. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

17. Life-Saving Treatments for Spinal Muscular Atrophy: Global Access and Availability.

18. Findings from the Longitudinal CINRG Becker Natural History Study.

19. Corneal confocal microscopy for identification of diabetic sensorimotor polyneuropathy: a pooled multinational consortium study

21. Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

27. Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy

30. A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial

33. Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy

34. Clinical, environmental, and genetic determinants of multiple sclerosis in children with acute demyelination: a prospective national cohort study

37. Corneal nerve and nerve conduction abnormalities in children with type 1 diabetes.

38. Machine learning classification of multiple sclerosis in children using optical coherence tomography.

39. Correction to: Impact of an electronic monitoring device and behavioural feedback on adherence to multiple sclerosis therapies in youth: results of a randomized trial

40. Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.

44. Validity of the diagnostic criteria for chronic cerebrospinal venous insufficiency and association with multiple sclerosis

46. Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial.

47. Routine lung volume recruitment in boys with Duchenne muscular dystrophy: a randomised clinical trial.

49. Clinical features and viral serologies in children with multiple sclerosis: a multinational observational study

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