First gene therapy trial targets deadly heart damage in rare neurological disease
NCT ID NCT05445323
Summary
This early-stage study is testing a new gene therapy, LX2006, for people with Friedreich's ataxia who have developed heart muscle damage. The therapy aims to deliver a healthy gene directly to heart cells to improve their function. Researchers will first check if the treatment is safe and look for signs it might help the heart work better.
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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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