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Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA). …

Friedreich AtaxiaCardiomyopathiesCardiac Hypertrophy+1 more

Weill Medical College of Cornell UniversityNCT05302271