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  1. Article

    Open Access

    Phase I clinical trial results of aceneuramic acid for GNE myopathy in Japan

    GNE myopathy (distal myopathy with rimmed vacuoles) is a rare intractable muscle disease caused by the mutations in GNE gene, with no therapeutic agents at present. The mutations in GNE (UDP-N-acetylglucosamine 2...

    Naoki Suzuki, Masaaki Kato, Hitoshi Warita… in Translational Medicine Communications (2018)

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