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  1. Article

    Open Access

    Myogenesis defects in a patient-derived iPSC model of hereditary GNE myopathy

    Hereditary muscle diseases are disabling disorders lacking effective treatments. UDP-N-acetylglucosamine-2-epimerase/N-acetylmannosamine kinase (GNE) myopathy (GNEM) is an autosomal recessive distal myopathy w...

    Rebecca E. Schmitt, Douglas Y. Smith IV, Dong Seong Cho in npj Regenerative Medicine (2022)