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Clinical trial

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)

NCT01784679 is registered in ClinicalTrials.gov with Ultragenyx Pharmaceutical Inc as lead sponsor.

NCT NCT01784679
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Registry record

As filed in ClinicalTrials.gov.

Title
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)
Lead sponsor
Ultragenyx Pharmaceutical Inc
Phase
Observational
Overall status
Completed
Start date
April 5, 2013
Completion date
November 30, 2017
Enrollment
319 (actual)
Sites
5

Conditions

As the registry lists them; a condition with its own page is linked.

distal myopathy with rimmed vacuoles (dmrv), gne myopathy, hereditary inclusion body myopathy, nonaka disease, quadriceps sparing myopathy (qsm)