Gene therapy hope for kids with rare gaucher disease
NCT ID NCT06528080
First seen Feb 04, 2026 · Last updated May 14, 2026 · Updated 14 times
Summary
This early-stage study tests a single injection of LY-M001 gene therapy in 9 children aged 6 to 17 with type 1 Gaucher disease. The goal is to see if it is safe and can help the body produce the missing enzyme. Researchers will monitor side effects and measure changes in enzyme levels and organ size over 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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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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