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    DRUG CLASS:

    Frataxin gene expression enhancer

    Related drugs:
    1m
    SUNRISE-FA: Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia (clinicaltrials.gov)
    P1/2, N=8, Active, not recruiting, Lexeo Therapeutics | Recruiting --> Active, not recruiting
    Enrollment closed • Gene therapy
    2ms
    Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia (clinicaltrials.gov)
    P1, N=25, Recruiting, Weill Medical College of Cornell University | N=10 --> 25
    Enrollment change • Gene therapy
    |
    AFP (Alpha-fetoprotein)
    |
    prednisone