Additional file 3: of Phase I clinical trial results of aceneuramic acid for GNE myopathy in Japan
Naoki Suzuki
Masaaki Kato
Hitoshi Warita
Rumiko Izumi
Maki Tateyama
Hiroshi Kuroda
Ryuta Asada
Akifumi Suzuki
Takuhiro Yamaguchi
Ichizo Nishino
Masashi Aoki
10.6084/m9.figshare.7056830.v1
https://springernature.figshare.com/articles/journal_contribution/Additional_file_3_of_Phase_I_clinical_trial_results_of_aceneuramic_acid_for_GNE_myopathy_in_Japan/7056830
Protocol for Trial 2 (in English). (DOC 500 kb)
2018-09-06 05:00:00
GNE myopathy
Aceneuramic acid
Phase I clinical trial
Ultra-orphan drug