Efficacy confirmation study of aceneuramic acid administration for GNE myopathy in Japan.
Madoka Mori-YoshimuraNaoki SuzukiMasahisa KatsunoMasanori P TakahashiSatoshi YamashitaYasushi OyaAtsushi HashizumeShinichiro YamadaMasayuki NakamoriRumiko IzumiMasaaki KatoHitoshi WaritaMaki TateyamaHiroshi KurodaRyuta AsadaTakuhiro YamaguchiIchizo NishinoMasashi AokiPublished in: Orphanet journal of rare diseases (2023)
ClinicalTrials.gov (NCT04671472).
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