54 results on '"Darras B"'
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2. P205 Impact of disease modifying treatment by three months of life on swallowing in spinal muscular atrophy type 1
3. P227 MANATEE: GYM329 (RO7204239) in combination with Risdiplam treatment in patients with spinal muscular atrophy (SMA)
4. P224 Effect of apitegromab on motor function at 36 months in patients with nonambulatory spinal muscular atrophy aged 2-12 years old
5. P234 Effect of Apitegromab on pedi-cat and promis-fatigue questionnaire at 36-months in patients with spinal muscular atrophy
6. P230 Safety update: Risdiplam clinical trial program for spinal muscular atrophy (SMA)
7. P.113 Safety update: Risdiplam clinical trial development program
8. P.102 Apitegromab in SMA: An analysis of multiple efficacy endpoints in the TOPAZ extension study
9. FP.23 Bulbar function for patients with spinal muscular atrophy type 1 following onasemnogene abeparvovec
10. O.01 Correlating molecular genetic testing for rare genetic variants with a broad clinicopathologic spectrum of congenital myopathies
11. VP.13 Updates on the development of the spinal muscular atrophy - person-reported outcome (SMA-PRO): A caregiver and self-proxy performance measure for child
12. FP.36 Genetic variants in DTNA cause a mild dominantly inherited muscular dystrophy
13. P.112 Impact of nusinersen on respiratory progression in paediatric patients with spinal muscular atrophy type 2 and non-ambulant type 3
14. P.100 Rationale/design of the phase 3b ASCEND study of investigational higher dose nusinersen in participants with SMA previously treated with risdiplam
15. P.109 FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam in Type 1 spinal muscular atrophy (SMA)
16. SMA - TREATMENT
17. SMA - TREATMENT
18. SMA CLINICAL DATA
19. SMA CLINICAL DATA
20. SMA – THERAPY
21. SMA – THERAPY
22. SMA - CLINICAL
23. SMA - CLINICAL
24. NEW GENES AND DISEASES / NGS & RELATED TECHNIQUES
25. SMA – THERAPY
26. SMA – THERAPY
27. SMA - CLINICAL
28. P.353FIREFISH Part 1: 16-month safety and exploratory outcomes of risdiplam (RG7916) treatment in infants with type 1 spinal muscular atrophy
29. P.221Trajectories of disease progression in ambulant and non ambulant SMA: 12 month follow-up
30. O.40Intrathecal administration of onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): phase 1/2a study (STRONG)
31. P.352Interim report on the safety and efficacy of longer-term treatment with nusinersen in later-onset spinal muscular atrophy (SMA): results from the SHINE study
32. P.270Association between plasma phosphorylated neurofilament heavy chain and efficacy endpoints in the nusinersen NURTURE study
33. P.220The revised Hammersmith scale (RHS) for spinal muscular atrophy: longitudinal trajectories in a large international cohort of patients with type 2 and 3 SMA
34. Sustainable Cooling and Lubrication Strategies in Machining Processes: A Comparative Study
35. SMA THERAPIES II AND BIOMARKERS
36. SMA THERAPIES II AND BIOMARKERS
37. DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY
38. SMA THERAPIES I
39. NEW GENES, FUNCTIONS AND BIOMARKERS
40. Sustainability Assessment of Machining with Nano-Cutting Fluids
41. Towards sustainability assessment of machining processes
42. Efficacy and safety of nusinersen in infants with spinal muscular atrophy (SMA): Final results from the phase 3 ENDEAR study
43. Interim analysis of the phase 3 CHERISH study evaluating nusinersen in patients with later-onset spinal muscular atrophy (SMA): Primary and descriptive secondary endpoints
44. Electrical impedance myography in Duchenne muscular dystrophy detects disease progression in boys younger than 7 years of age
45. Revised Hammersmith scale for spinal muscular atrophy: Longitudinal changes over six and twelve months in a large international cohort
46. Should motor function determine the timing of scoliosis surgery in spinal muscular atrophy?
47. Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials
48. G.O.18
49. O.9 First-in-human phase I study to assess safety, tolerability and dose for intrathecal injection of ISIS-SMNRx in SMA patients
50. G.P.2.01 The natural history of spinal muscular atrophy – preliminary results from the PNCR network
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