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Ultragenyx Pharmaceutical Inc

Clinical trials sponsored by Ultragenyx Pharmaceutical Inc, explained in plain language.

Ultragenyx Pharmaceutical Inc RARE NASDAQ

Biopharmaceutical company developing treatments for rare and ultra-rare diseases.

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Not investment advice. Company information is shown for context only. Clinical trials frequently fail.

Trials to join now! 6 Not yet recruiting 1 Not yet finished but already full! 13 Completed 3

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.

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

Last updated Jun 27, 2026 12:04 UTC