Gene therapy injection aims to tame rare enzyme disorder in kids
NCT ID NCT06528080
First seen Feb 04, 2026 · Last updated May 22, 2026 · Updated 16 times
Summary
This early-stage study tests a single intravenous dose of LY-M001 in 9 children (ages 6 to 17) with type 1 Gaucher disease. The goal is to see if the treatment is safe and can restore the missing enzyme, potentially reducing the need for lifelong enzyme replacement therapy. Researchers will monitor side effects and measure enzyme activity in the blood for up to a year.
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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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Contacts and locations
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Locations
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Shanghai Children's Medical Center, School of Medicine, Shanghai Jiao Tong University
Shanghai, Shanghai Municipality, China
Conditions
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