First Huntington’s patient dosed in large trial of oral therapy votoplam

Publication date: May 13, 2026

In particular, the evidence of dose-dependent slowing of progression on the cUHDRS disease rating scale in the Stage 2 study participants supports the Novartis-initiated Phase 3 INVEST-HD study. We look forward to continuing to review the data and aligning on potential regulatory interactions based on the results with our partner Novartis.

Concepts Keywords
Ceo Clinical
Huntingtons Dose
Nfl Hd
Proteinhuntingtons Huntingtons
Yearlong Novartis
Participants
Phase
Placebo
Progression
Ptc
Slowing
Stage
Study
Trial
Votoplam

Semantics

Type Source Name
drug DRUGBANK Tropicamide
drug DRUGBANK Methionine
disease MESH disease progression
disease MESH PTC
pathway REACTOME Release

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