GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
Conditions
- Hereditary Inclusion Body Myopathy
- GNE Myopathy
- Nonaka Disease
- Quadriceps Sparing Myopathy (QSM)
- Distal Myopathy With Rimmed Vacuoles (DMRV)
Sponsor
Ultragenyx Pharmaceutical Inc
Collaborators