- Conditions
- Hereditary Inclusion Body Myopathy, GNE Myopathy, Nonaka Disease, Quadriceps Sparing Myopathy (QSM), Distal Myopathy With Rimmed Vacuoles (DMRV)
- Interventions
- Not listed
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 319 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 1
- States / cities
- Irvine, California
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Showing 1–3
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- Conditions
- GNE Myopathy, GNE Related Diseases
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 4 Years to 80 Years
- Enrollment
- 78 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2011
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- GNE Myopathy, Hereditary Inclusion Body Myopathy
- Interventions
- Sialic Acid Extended Release (SA-ER), Placebo
- Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 3
- States / cities
- Los Angeles, California • St Louis, Missouri • New York, New York