Safety and efficacy of aceneuramic acid in GNE myopathy: open-label extension study.
Naoki SuzukiMadoka Mori-YoshimuraMasahisa KatsunoMasanori P TakahashiSatoshi YamashitaYasushi OyaAtsushi HashizumeShinichiro YamadaMasayuki NakamoriRumiko IzumiMasaaki KatoHitoshi WaritaMaki TateyamaHiroshi KurodaRyuta AsadaTakuhiro YamaguchiIchizo NishinoMasashi AokiPublished in: Journal of neurology, neurosurgery, and psychiatry (2024)