risdiplam

Trials studying risdiplam

• phase4Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)active not recruitingn=403
• phase4A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapyrecruitingn=28
• phase2A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophyactive not recruitingn=26
• phase4A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapyrecruitingn=28
• phase2A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophyrecruitingn=10
• phase2A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophyrecruitingn=52
• phase3A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophyactive not recruitingn=259
• —Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restorationcompletedn=18