One-Time gene shot aims to ease wilson disease burden
NCT ID NCT06650319
First seen Jan 27, 2026 · Last updated May 15, 2026 · Updated 15 times
Summary
This early-phase trial tests a single-dose gene therapy (LY-M003) in 18 adults and children with Wilson disease, a rare genetic condition causing copper buildup. The treatment delivers a working gene to liver cells to help remove excess copper. The main goals are checking safety and seeing if patients can reduce their daily medications.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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First Affiliated Hospital of Zhejiang University
RECRUITINGHangzhou, Zhejiang, 312000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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