Pharmacokinetic Study on N-acetylneuraminic Acid

计划入组 6 人 · 首次发布于 2010 年 11 月 9 日

Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

计划入组 89 人 · 首次发布于 2015 年 3 月 4 日

Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

计划入组 143 人 · 首次发布于 2016 年 4 月 13 日

myopathy program

AAV-MTM1

autologous myogenic stem cell therapy (mitochondrial myopathy)

MONIKA JOSHI

India

TANAKA HIROAKI

Japan

OSAKA UNIVERSITY

Japan

FDA Clears Ultragenyx IND Application for UX016 to Treat Rare GNE Myopathy

Mavodelpar Shows Promise in Phase 1 Trial for Primary Mitochondrial Myopathy

Solve GNE Advances First Gene Therapy for GNE Myopathy Toward Phase I Clinical Trial