Efficacy confirmation study of aceneuramic acid administration for GNE myopathy in Japan

BACKGROUND: A rare muscle disease, GNE myopathy is caused by mutations in the GNE gene involved in sialic acid biosynthesis. Our recent phase II/III study has indicated that oral administration of aceneuramic acid to patients slows disease progression. METHODS: We conducted a phase III, randomized,...

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Detalles Bibliográficos
Autores principales: Mori-Yoshimura, Madoka, Suzuki, Naoki, Katsuno, Masahisa, Takahashi, Masanori P., Yamashita, Satoshi, Oya, Yasushi, Hashizume, Atsushi, Yamada, Shinichiro, Nakamori, Masayuki, Izumi, Rumiko, Kato, Masaaki, Warita, Hitoshi, Tateyama, Maki, Kuroda, Hiroshi, Asada, Ryuta, Yamaguchi, Takuhiro, Nishino, Ichizo, Aoki, Masashi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10416530/
https://www.ncbi.nlm.nih.gov/pubmed/37568154
http://dx.doi.org/10.1186/s13023-023-02850-y

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