Investigations Into ISCU Myopathy or Iron Sulfur Scaffold U Protein Myopathy

2 participants · First posted Mar 8, 2012

Myotubular Myopathy Event Study

33 participants · First posted Apr 26, 2013

Metabolic Myopathy in Endocrinopathy

1 participants · First posted Jul 13, 2022

myopathy program

AAV-MTM1

autologous myogenic stem cell therapy (mitochondrial myopathy)

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

Mavodelpar Shows Promise in Phase 1 Trial for Primary Mitochondrial Myopathy

RESTEM's Restem-L Receives FDA Fast Track Designation for Idiopathic Inflammatory Myopathy