Studies
A randomized, placebo-controlled, double-blind Phase 3 study to evaluate the efficacy, safety and tolerability of votoplam in participants with Huntingtons Disease
Neurology
Adult Subjects
The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control arm.
HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.
A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, DOSE-FINDING STUDY TO EVALUATE THE SAFETY, BIOMARKERS, AND EFFICACY OF TOMINERSEN IN INDIVIDUALS WITH PRODROMAL AND EARLY MANIFEST HUNTINGTONS DISEASE
Neurology
Adult Subjects
This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease (HD).
Parkinsons Foundation PD GENEration Genetic Registry
Neurology
Adult Subjects
Female Subjects
Male Subjects