Clinical Trial Update: Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 [Phase 3]

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Summary

A Phase 3 trial of a single-dose gene replacement therapy for Spinal Muscular Atrophy Type 1 has completed, with results posted in 2021. The sponsor is Novartis…

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Diseases / indications

• Spinal Muscular Atrophy

Drugs mentioned

• Zolgensma (onasemnogene abeparvovec)

Snippet

Sponsor: Novartis Gene Therapies | Conditions: SMA | Phase 3 | Status: COMPLETED | Results posted: 2021-04-02