branaplam

DRUG2 trials

Sponsors

Novartis Pharmaceuticals

Conditions

Early Manifest Huntington DiseaseSpinal Muscular Atrophy

Phase 1

An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)

CompletedNCT02268552

Novartis PharmaceuticalsSpinal Muscular Atrophy

Start: 2015-04-02End: 2022-12-29Updated: 2025-04-09

Phase 2

A Dose Range Finding Study With Open-Label Extension to Evaluate the Safety of Oral LMI070/Branaplam in Early Manifest Huntington's Disease

TerminatedNCT05111249

Novartis PharmaceuticalsEarly Manifest Huntington Disease

Start: 2021-12-08End: 2023-10-27Updated: 2025-05-16

Related Papers

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An alternative splicing modulator decreases mutant HTT and improves the molecular fingerprint in Huntington's disease patient neurons.

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An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion.

2022-03-0388 citations