One-Shot gene therapy tested in kids with rare genetic disorder
NCT ID NCT06528080
Summary
This early-stage study is testing a single intravenous infusion of an experimental gene therapy called LY-M001 in children and adolescents with Gaucher disease type 1. The main goal is to check if the treatment is safe and tolerable. Researchers will also look for early signs that it might help control the disease by measuring enzyme activity and symptoms like liver and spleen size.
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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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