GNE MYOPATHY

Clinical trials for GNE MYOPATHY explained in plain language.

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Not yet recruiting 1 Completed 1

New drug UX016 targets rare muscle disease

Disease control Not yet recruiting

This early-stage trial tests UX016, a drug designed to help people with GNE myopathy, a rare genetic muscle-weakening disease. Researchers will give the drug or a placebo to 24 adults to see if it is safe and improves muscle strength. The study is not yet recruiting.

Matched conditions: GNE MYOPATHY

Phase: PHASE1, PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control

Last updated Jun 27, 2026 12:04 UTC

GNE MYOPATHY

Get alerted when new GNE MYOPATHY trials appear

New drug UX016 targets rare muscle disease