Safety and efficacy of aceneuramic acid in GNE myopathy: open-label extension study-Reference-Cited by-同舟云学术

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Safety and efficacy of aceneuramic acid in GNE myopathy: open-label extension study

Published:2024-06-05 Issue: Volume: Page:jnnp-2024-333853
ISSN:0022-3050
Container-title:Journal of Neurology, Neurosurgery & Psychiatry
language:en
Short-container-title:J Neurol Neurosurg Psychiatry

Author:

Suzuki Naoki^ORCID,Mori-Yoshimura Madoka^ORCID,Katsuno Masahisa^ORCID,Takahashi Masanori P^ORCID,Yamashita Satoshi^ORCID,Oya Yasushi,Hashizume Atsushi,Yamada Shinichiro,Nakamori Masayuki,Izumi Rumiko,Kato Masaaki,Warita Hitoshi,Tateyama Maki,Kuroda Hiroshi,Asada Ryuta,Yamaguchi Takuhiro,Nishino Ichizo^ORCID,Aoki Masashi

Funder

Japan Agency for Medical Research and Development

National Institutes of Biomedical Innovation, Health and Nutrition

Publisher

BMJ

Reference5 articles.

1. Recent advances in establishing a cure for GNE myopathy;Yoshioka;Curr Opin Neurol,2022

2. The UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase gene is mutated in recessive hereditary inclusion body myopathy

3. Phase II/III study of Aceneuramic acid administration for GNE myopathy in Japan;Suzuki;J Neuromuscul Dis,2023

4. Efficacy confirmation study of Aceneuramic acid administration for GNE myopathy in Japan;Mori-Yoshimura;Orphanet J Rare Dis,2023

5. Aceneuramic acid extended release administration maintains upper limb muscle strength in a 48-week study of subjects with GNE myopathy: results from a phase 2, randomized, controlled study;Argov;J Neuromuscul Dis,2016

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