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

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Summary

Phase 3 trial results for a gene replacement therapy in spinal muscular atrophy type 1 were completed, with results posted in July 2020. The program is develope…

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Diseases / indications

• Spinal Muscular Atrophy

Drugs mentioned

• Zolgensma (onasemnogene abeparvovec)

Snippet

Sponsor: Novartis Gene Therapies | Conditions: SMA - Spinal Muscular Atrophy, Gene Therapy | Phase 3 | Status: COMPLETED | Results posted: 2020-07-16