Friedreich’s ataxia is the most common inherited ataxia in humans and results from a deficiency of the mitochondrial protein research hub

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects with Friedreich’s Ataxia

This study is testing a new medicine, Nomlabofusp, for Friedreich’s Ataxia. It’s comparing it to a dummy treatment over 72 weeks to see if it helps improve symptoms and is safe. The goal is to find better ways to manage this rare condition.