Gene therapy aims to fix heart damage in rare nerve disease
NCT ID NCT05302271
First seen Jan 03, 2026 · Last updated May 22, 2026 · Updated 18 times
Summary
This early-stage study tests a gene therapy called AAVrh.10hFXN for heart problems caused by Friedreich's ataxia, a rare genetic disorder. The therapy delivers a working gene to help heart cells function better. About 25 people aged 12 to 50 with mild to moderate heart disease will receive a single IV dose, and researchers will monitor safety for 5 years.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Weill Cornell Medicine
RECRUITINGNew York, New York, 10021, United States
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