Clinical Trial Update: Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA) [Phase 3]

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Summary

A Phase 3 trial evaluated the efficacy and safety of intrathecal AVXS-101 in pediatric patients with spinal muscular atrophy, with results posted after study co…

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Diseases / indications

• Spinal Muscular Atrophy

Drugs mentioned

• AVXS-101 (onasemnogene abeparvovec)

Snippet

Sponsor: Novartis Pharmaceuticals | Conditions: Type 2 Spinal Muscular Atrophy | Phase 3 | Status: COMPLETED | Results posted: 2025-12-08