Gene therapy targets heart damage in rare neurological disease
NCT ID NCT05445323
First seen Jan 04, 2026 · Last updated May 15, 2026 · Updated 15 times
Summary
This early-stage study tests a gene therapy called LX2006 in 8 people with Friedreich's ataxia who also have heart muscle disease. The treatment delivers a working copy of the frataxin gene to heart cells through a single IV infusion. Researchers will monitor safety and heart function for up to 5 years to see if the therapy can slow or improve heart damage.
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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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Contacts and locations
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Locations
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Ataxia Center and HD Center of Excellence, University of California
Los Angeles, California, 90095, United States
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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University of South Florida
Tampa, Florida, 33612, United States
Conditions
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