BARTH SYNDROME
Clinical trials for BARTH SYNDROME explained in plain language.
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New shot aims to help kids with rare heart-muscle disease move easier
Disease control Recruiting nowThis 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…
Matched conditions: BARTH SYNDROME
Phase: PHASE4 • Sponsor: Stealth BioTherapeutics Inc. • Aim: Disease control
Last updated Jul 15, 2026 00:00 UTC
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Worldwide data pool could pave the way for mitochondrial disease trials
Knowledge-focused Recruiting nowThis study creates a global registry for people with mitochondrial disorders—rare diseases that affect energy production in cells. By collecting health data from 6,000 participants worldwide, researchers aim to understand how these diseases progress and identify the best ways to …
Matched conditions: BARTH SYNDROME
Sponsor: LMU Klinikum • Aim: Knowledge-focused
Last updated Aug 09, 2026 00:00 UTC