New shot aims to strengthen muscles in rare genetic disease
Disease control
Not yet recruiting
This study tests a daily injection called elamipretide in 48 people with genetically confirmed Barth syndrome, a rare genetic condition that causes muscle weakness and heart problems. The goal is to see if the drug improves muscle strength and mobility over 72 weeks compared to a…
Phase: PHASE4 • Sponsor: Stealth BioTherapeutics Inc. • Aim: Disease control
Last updated May 06, 2026 16:14 UTC