Experimental drug aims to halt rare neurological decline
Disease control
Not yet recruiting
This Phase 3 trial tests whether vatiquinone can slow the progression of Friedreich's ataxia, a rare genetic disorder that damages the nervous system and impairs movement. About 120 adults with moderate symptoms will take the drug for 24 months. Researchers will measure changes i…
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jul 04, 2026 00:00 UTC