Clinical Trial Update: Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies [Phase 3]

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Summary

Phase 3 trial of a single-dose gene replacement therapy for spinal muscular atrophy type I with 1-2 SMN2 copies has completed and posted results.

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Diseases / indications

• Spinal Muscular Atrophy

Drugs mentioned

• Zolgensma (onasemnogene abeparvovec)

Snippet

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