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