356 results on '"Mah, Jean"'
Search Results
2. Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab
3. Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy
4. Macrostructural Brain Abnormalities in Spinal Muscular Atrophy: A Case-Control Study.
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
10. The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review
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
14. Early onset facioscapulohumeral dystrophy – a systematic review using individual patient data
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
20. Can we have an overall osteoarthritis severity score for the patellofemoral joint using magnetic resonance imaging? Reliability and validity
21. Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function
22. Disease-specific and glucocorticoid-responsive serum biomarkers for Duchenne Muscular Dystrophy
23. Early corneal nerve fibre damage and increased Langerhans cell density in children with type 1 diabetes mellitus
24. Alberta Spinal Muscular Atrophy Newborn Screening—Results from Year 1 Pilot Project.
25. Current Cardiac Imaging Approaches in Duchenne Muscular Dystrophy
26. A comprehensive literature review on hypothermia and early extubation following coronary artery bypass surgery
27. Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy
28. A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy
29. Current and Emerging Therapies for Duchenne Muscular Dystrophy
30. A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial
31. An Overview of Congenital Myopathies
32. Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study.
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
35. Parents' Global Rating of Mental Health Correlates with SF-36 Scores and Health Services Satisfaction
36. Understanding the experiences of lung volume recruitment among boys with Duchenne muscular dystrophy: A multicenter qualitative 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.
41. Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patients
42. Being the lifeline: The parent experience of caring for a child with neuromuscular disease on home mechanical ventilation
43. Neurodegeneration in D-bifunctional protein deficiency: diagnostic clues and natural history using serial magnetic resonance imaging
44. Validity of the diagnostic criteria for chronic cerebrospinal venous insufficiency and association with multiple sclerosis
45. Motor unit number estimations are smaller in children with type 1 diabetes mellitus: A case–cohort study
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.
48. Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
49. Clinical features and viral serologies in children with multiple sclerosis: a multinational observational study
50. Respiratory Management Strategies for Duchenne Muscular Dystrophy: Practice Variation Amongst Canadian Sub-Specialists
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