Phase II/III Study of Aceneuramic Acid Administration for GNE Myopathy in Japan

BACKGROUND: GNE myopathy is an ultra-rare muscle disease characterized by a reduction in the synthesis of sialic acid derived from pathogenic variants in the GNE gene. No treatment has been established so far. OBJECTIVE: We evaluated the safety and efficacy of oral supplementation of aceneuramic aci...

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Detalles Bibliográficos
Autores principales: Suzuki, Naoki, Mori-Yoshimura, Madoka, 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: IOS Press 2023
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10357206/
https://www.ncbi.nlm.nih.gov/pubmed/37125562
http://dx.doi.org/10.3233/JND-230029

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