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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: Recruiting

First-in-human Study of UX016 in GNEM

The goal of this study is to assess the safety and dose of UX016 and its impact 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: Oct 13, 2026Locations: 2
Eligibility criteria

A confirmed diagnosis of GNEM (also known as distal myopathy with rimmed vacuole... [+5]

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

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