Phase I clinical trial results of aceneuramic acid for GNE myopathy in Japan

Phase I clinical trial results of aceneuramic acid for GNE myopathy in Japan

Abstract Background GNE myopathy (distal myopathy with rimmed vacuoles) is a rare intractable muscle disease caused by the mutations in GNE gene, with no therapeutic agents at present. The mutations in GNE (UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase) gene result in a deficiency o...

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Bibliographic Details
Main Authors: Naoki Suzuki, Masaaki Kato, Hitoshi Warita, Rumiko Izumi, Maki Tateyama, Hiroshi Kuroda, Ryuta Asada, Akifumi Suzuki, Takuhiro Yamaguchi, Ichizo Nishino, Masashi Aoki
Format: Article
Language:English
Published: BMC 2018-09-01
Series:Translational Medicine Communications
Subjects:
GNE myopathy
Aceneuramic acid
Phase I clinical trial
Ultra-orphan drug
Online Access:http://link.springer.com/article/10.1186/s41231-018-0025-0

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http://link.springer.com/article/10.1186/s41231-018-0025-0

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