Clinical Trial Update: Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 [Phase 3]

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Summary

Phase 3 trial update evaluating intravenous Zolgensma (Onasemnogene abeparvovec-xioi) in presymptomatic spinal muscular atrophy patients with multiple SMN2 copi…

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Diseases / indications

• Spinal Muscular Atrophy

Drugs mentioned

• Zolgensma (onasemnogene abeparvovec)

Snippet

Sponsor: Novartis Gene Therapies | Conditions: Spinal Muscular Atrophy | Phase 3 | Status: COMPLETED | Results posted: 2022-01-11