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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 Not yet recruiting

    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…

    Matched conditions: BARTH SYNDROME

    Phase: PHASE4 • Sponsor: Stealth BioTherapeutics Inc. • Aim: Disease control

    Last updated Jun 27, 2026 12:29 UTC

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