832 results on '"Servais, Laurent"'
Search Results
2. Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study.
3. Quantitative measures of motor development in Angelman syndrome
4. Author Correction: miR-379 links glucocorticoid treatment with mitochondrial response in Duchenne muscular dystrophy
5. Clinical and genetic features of patients suffering from CMT4J
6. DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials.
7. Quantitative proteome analysis of LAP1-deficient human fibroblasts: A pilot approach for predicting the signaling pathways deregulated in LAP1-associated diseases
8. 2024 update: European consensus statement on gene therapy for spinal muscular atrophy
9. Thrombotic Microangiopathy as an Emerging Complication of Viral Vector–Based Gene Therapy
10. Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in England
11. Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec
12. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
13. The use of digital outcome measures in clinical trials in rare neurological diseases: a systematic literature review
14. Evolving regulatory perspectives on digital health technologies for medicinal product development
15. Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA)
16. Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom
17. A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders
18. Cost-effectiveness of spinal muscular atrophy newborn screening based on real-world data in Belgium
19. First regulatory qualification of a digital primary endpoint to measure treatment efficacy in DMD
20. Use of MFM-20 to monitor SMA types 1 and 2 patients treated with nusinersen
21. Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
22. Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
23. Correction to: Two‑year efficacy and safety of risdiplam in patients with type 2 or non‑ambulant type 3 spinal muscular atrophy (SMA)
24. Spinal Muscular Atrophy Update in Best Practices
25. 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.
26. Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping
27. Emergencies in Motor Neuron Diseases
28. Leveraging Natural History Data in One- and Two-Arm Hierarchical Bayesian Studies of Rare Disease Progression
29. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial
30. Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial
31. Patients’ Perceptions of Nusinersen Effects According to Their Responder Status
32. Relationship Between Hand Strength and Function in Duchenne Muscular Dystrophy and Spinal Muscular Atrophy: Implications for Clinical Trials
33. Stride Velocity 95th Centile Detects Decline in Ambulatory Function Over Shorter Intervals than the 6-Minute Walk Test or North Star Ambulatory Assessment in Duchenne Muscular Dystrophy
34. Digital Outcome Measure Assessing the Motor Function of Patients with Amyotrophic Lateral Sclerosis (P5-11.011)
35. RAINBOWFISH: Primary Efficacy and Safety Data in Risdiplam-treated Infants with Presymptomatic Spinal Muscular Atrophy (SMA) (S37.006)
36. Reliability and External Validity of Digital Passive Gait Tracking in MS (P6-6.008)
37. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
38. Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial
39. Emerging therapies for Duchenne muscular dystrophy
40. Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls
41. Severe ACTA1-related nemaline myopathy: intranuclear rods, cytoplasmic bodies, and enlarged perinuclear space as characteristic pathological features on muscle biopsies
42. 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
43. Impact of Disease Severity and Disease-Modifying Therapies on Myostatin Levels in SMA Patients.
44. Feasibility and Acceptability of a Newborn Screening Program Using Targeted Next-Generation Sequencing in One Maternity Hospital in Southern Belgium.
45. Scoring People With Spinal Muscular Atrophy on the Motor Function Measure Using the Microsoft Kinect
46. A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy
47. The Importance of Early Treatment of Inherited Neuromuscular Conditions
48. Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry
49. Newborn screening of neuromuscular diseases
50. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial
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