Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
A Phase 1 Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ManNAc in Subjects With Primary Podocyte Diseases
|3||Active, not recruiting||
An Open Label Phase 2 Study of DEX-M74 in Subjects With GNE Myopathy
A Natural History Study of Patients With GNE Myopathy
† Study has passed its completion date and status has not been verified in more than two years.