Health Trials

GNE Myopathy

1

Review clinical trials related to GNE Myopathy. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Not yet recruiting

First-in-human Study of UX016 in GNEM

The main goal of this study is to evaluate the safety of UX016 and to evaluate the impact of UX016 on muscle strength in adults with GNE Myopathy (GNEM).

Participants needed: 24
Trial details
Phase: Phase 1, Phase 2Age: 18-55Biological sex: AllType: InterventionalSponsor: Ultragenyx Pharmaceutical IncUpdated: Apr 6, 2026Locations: 2
Eligibility criteria

A confirmed diagnosis of GNEM (also known as hereditary inclusion body myopathy... [+4]

Ingestion of N-acetyl-D-mannosamine (ManNAc), SA, or related metabolites, includ... [+10]

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