Gene therapy targets deadly heart damage in rare neurological disease
NCT ID NCT05445323
Summary
This early-stage trial is testing whether a single dose of LX2006 gene therapy can safely treat heart muscle damage in people with Friedreich's ataxia. The therapy delivers a healthy gene directly to heart cells to improve their function. Eight participants with confirmed Friedreich's ataxia and heart involvement will be followed for five years to check safety and whether heart function improves.
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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
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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