New shot aims to help kids with rare heart-muscle disease move easier
Disease control
Recruiting now
This study tests a daily injection called elamipretide in 48 people with genetically confirmed Barth syndrome, a rare condition that causes muscle weakness and heart problems. Participants will receive either the drug or a placebo for 72 weeks. The main goal is to see if the drug…
Phase: PHASE4 • Sponsor: Stealth BioTherapeutics Inc. • Aim: Disease control
Last updated Jul 15, 2026 00:00 UTC