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…
Phase: PHASE1, PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 17, 2026 05:54 UTC