First-in-Human gene therapy trial targets devastating heart condition
NCT ID NCT05302271
First seen Jan 03, 2026 · Last updated Apr 09, 2026 · Updated 16 times
Summary
This is an early-stage study testing the safety of a new gene therapy for the heart damage caused by Friedreich's ataxia. The therapy uses a modified virus to deliver a healthy gene directly to the heart. Researchers will enroll 25 participants to find a safe dose and look for early signs that it might help.
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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.
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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Locations
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Weill Cornell Medicine
RECRUITINGNew York, New York, 10021, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Conditions
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