A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy (NCT03779334)

🧪 Interventions in This Study

• Risdiplam (DRUG)

Full eligibility criteria are available on the official NIH protocol. View the criteria ↗

📍 All Participating Trial Locations (7 Sites)

• Nemours Children's Hospital — Orlando, Florida
• Sydney Children's Hospital — Randwick, New South Wales
• Chr de La Citadelle — Liège,
• Hospital das Clinicas - FMUSP_X — São Paulo, São Paulo
• Szpital Gdanskiego Uniwersytetu Medycznego — Gda?sk,
• Russian Children Neuromuscular Center of Veltischev — Moscow, Moscow Oblast
• Kaohsiung Medical University Chung-Ho Hospital — Kaohsiung City,

💰 Cost, Insurance & Patient Rights

• 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
• Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
• Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

More studies in Muscular Atrophy, Spinal

• A 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 Therapy — Little Rock, Arkansas
• A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy — Little Rock, Arkansas
• A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK) — Orange, California
• A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec — Flower Mound, Texas
• A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies — Palo Alto, California

Explore by condition: All Muscular Atrophy, Spinal clinical trials

💡 How to Participate

1. Check the checklist above to see if you may qualify.
2. Review the official NIH record below to find the coordinating site.
3. Ask about the visit schedule, what is covered at no cost, and any travel support.
4. Confirm with your own doctor before making a decision.