224 results on '"Gidaro, T"'
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2. Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping
3. Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy
4. P.110 JEWELFISH: 24-month safety and pharmacodynamic data in non-treatment-naïve patients with spinal muscular atrophy (SMA)
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. Targeted exomes reveal simultaneous MFN2 and GDAP1 mutations in a severe Charcot‐Marie‐Tooth disease type 2 phenotype
7. Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients
8. 238P NMD670, a first-in-class skeletal muscle ClC-1 Inhibitor in myasthenia gravis: the SYNAPSE-MG dose-finding study.
9. 103P NMD670, a first-in-class skeletal muscle ClC-1 Inhibitor in Charcot-Marie-Tooth disease: the SYNAPSE-CMT phase 2 study.
10. CLINICAL RESEARCH
11. Normalized grip strength is a sensitive outcome measure through all stages of Duchenne muscular dystrophy
12. DMD – BIOMARKERS & OUTCOME MEASURES
13. Clinical changes over time in patients with centronuclear myopathy due to mutations in DNM2 gene enrolled in a European prospective natural history study
14. The mitotic clock in skeletal muscle regeneration, disease and cell mediated gene therapy
15. Treatment with Ataluren for Duchene Muscular Dystrophy
16. Sitting in patients with spinal muscular atrophy type 1 treated with nusinersen
17. Baseline characteristics of patients with centronuclear myopathy due to mutations in DNM2 gene enrolled in a European prospective natural history study
18. SMA Clinical Data, Outcome Measures and Registries : P.88Associations between NMR, electrophysiological, strength and function variables in SMA type 2 and 3
19. P.107Clinical changes over time in patients with centronuclear myopathy due to mutations in DNM2 gene enrolled in a European prospective natural history study
20. P.200Feasibility and baseline values of continuous movement measurement in patients with centronuclear myopathy by using ActiMyo®
21. P.240ASC-1 related myopathy: phenotypic spectrum and pathophysiology of an emerging congenital myopathy
22. O.28Safety and tolerability of suvodirsen (WVE-210201) in patients with Duchenne muscular dystrophy: results from a phase 1 clinical trial
23. P.191Maximal stride velocity detects positive and negative changes over 6- month-time period in ambulant patients with Duchenne muscular dystrophy
24. OUTCOME MEASURES
25. MRI – MUSCLE IMAGING
26. CLINICAL RESEARCH: O.7 Prospective natural history of upper limb disease evolution in Duchenne muscular dystrophy
27. DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY
28. DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY
29. CONGENITAL MYASTHENIC SYNDROMES AND MYASTHENIA
30. REGISTRIES AND CARE OF NEUROMUSCULAR DISORDERS
31. SMA CLINICAL DATA, OUTCOME MEASURES AND REGISTRIES
32. CONGENITAL MYOPATHIES: GENERAL AND RYR1
33. SMA THERAPIES I
34. Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy
35. GNE-myopathy (HIBM): Upper and lower extremity muscle strength declines over time in a prospective study
36. Activities of daily living detection using home activity monitoring device in Duchenne muscular dystrophy patients
37. Innovative home activity monitoring in non-ambulant patients with spinal muscular atrophy: a multicenter observational trial
38. Longitudinal home-monitoring data in non-ambulant patients with Duchenne muscular atrophy
39. New myotubular myopathy classification
40. Associations between NMR, electrophysiological, strength and function variables in SMA type 2 and 3
41. Safety, tolerability and clinical efficacy of nusinersen in SMA type 1 older than 7 months: a prospective study
42. Results of a Phase 1b/2 Study of ATYR1940 in adolescents and young adults with early onset facioscapulohumeral muscular dystrophy (FSHD) (ATYR1940-C-003)
43. ActiMyo home monitoring in adult patients with limb girdle muscular dystrophy type 2B and facioscapulohumeral muscular dystrophy in study ATYR 1940-C-004
44. First experience of Nusinersen early access program in patients with spinal muscular atrophy type 1
45. Rimeporide: safety, tolerability and pharmacokinetic results from a phase Ib study in DMD boys as well as exploratory biomarkers
46. Longitudinal data of the European prospective natural history study of patients with type 2 and 3 spinal muscular atrophy
47. Longitudinal data of patients with myotubular myopathy enrolled in a European prospective and longitudinal natural history study
48. Characterization of pulmonary function in 10–18 year old patients with Duchenne muscular dystrophy
49. Targeted exomes reveal simultaneousMFN2andGDAP1mutations in a severe Charcot-Marie-Tooth disease type 2 phenotype
50. An international prospective, longitudinal study of the natural history and functional status of patients with myotubular myopathy
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