New drug UX016 targets rare muscle disease
NCT ID NCT07511556
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
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.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- UX016 (Sialic Acid-C16 prodrug)
- What this could lead to
- If successful, UX016 could become the first treatment to slow or improve muscle weakness in people with GNE myopathy.
- What could go wrong
- This is a very early, small trial (24 people) testing safety and effectiveness. It may not show benefit, and side effects are unknown.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
Locations
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Clinical Trial Site
Orange, California, 92868, United States
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Clinical Trial Site
Iselin, New Jersey, 08830, United States
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