One-Shot gene therapy could end lifelong injections for gaucher disease
NCT ID NCT07223944
Summary
This study is testing a one-time gene therapy called FLT201 for adults with Gaucher disease type 1. The goal is to see if this single treatment can replace the need for lifelong, regular enzyme replacement therapy. Researchers will check if the therapy is safe and if it keeps patients' blood counts stable for a year after stopping their standard treatments.
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