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BioWorld - Thursday, March 26, 2026
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Home » EPI-321 gene therapy leads phenotypic rescue in humanized murine model of FSHD
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Neurology/Psychiatric

EPI-321 gene therapy leads phenotypic rescue in humanized murine model of FSHD

March 24, 2023
Facioscapulohumeral muscular dystrophy (FSHD) is a skeletal muscular dystrophy characterized by DNA hypomethylation of D4Z4 repeat units of a macrosatellite array found at the distal end of chromosome region 4q35, which causes a myotoxic expression of DUX4. Researchers from Epic-Bio presented the discovery of EPI-321, a novel gene therapy candidate for the treatment of FSHD.
BioWorld Science Conferences Neurology/psychiatric

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