GNE MYOPATHY

Clinical trials for GNE MYOPATHY explained in plain language.

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Not yet recruiting 1 Completed 1

New drug UX016 aims to strengthen muscles in rare disease

Disease control Not yet recruiting

This study tests a new drug called UX016 in 24 adults with GNE myopathy, a rare muscle-weakening disease. The main goals are to check if the drug is safe and if it can improve muscle strength. Participants will receive either UX016 or a placebo, and researchers will measure chang…

Matched conditions: GNE MYOPATHY

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

Last updated May 17, 2026 09:37 UTC

GNE MYOPATHY

Get alerted when new GNE MYOPATHY trials appear

New drug UX016 aims to strengthen muscles in rare disease