One-Shot gene therapy could end lifelong injections for gaucher patients
NCT ID NCT07223944
Summary
This Phase 3 trial is testing whether a single gene therapy called FLT201 can safely and effectively treat adults with Gaucher disease type 1. The goal is to replace the need for lifelong, regular enzyme replacement therapy (ERT) or substrate reduction therapy (SRT). Participants will receive FLT201 and then stop their current treatments to see if the gene therapy alone can control the disease.
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