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Gene Therapy
Gene Therapy for Cardiomyopathy in Friedreich's Ataxia
Recruiting1 awardPhase 1 & 2
Los Angeles, California
This trial will test a new gene therapy for Friedreich's Ataxia, a rare degenerative disease. The therapy will be delivered intravenously and will be evaluated for safety and efficacy over a 5 year period.
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Get in touch with the study teamWe’ll send your responses to the study team and help you to setup your first screening. This is not the start of informed consent. Screening happens between you and the trial.