Clinical Trial Update: An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) [Phase 1, Phase 2]

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Summary

An open-label Phase 1/2 study of branaplam (LMI070) in patients with type 1 spinal muscular atrophy has completed, with results posted.

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Diseases / indications

• Spinal Muscular Atrophy

Drugs mentioned

• Branaplam (branaplam)

Snippet

Sponsor: Novartis Pharmaceuticals | Conditions: Spinal Muscular Atrophy | Phase 1, Phase 2 | Status: COMPLETED | Results posted: 2025-04-09