Gene therapy trial offers hope for rare genetic disorder
NCT ID NCT07223944
Summary
This study is testing whether a one-time gene therapy called FLT201 can safely control Gaucher disease type 1 in adults. Participants will receive the gene therapy and then stop their current enzyme replacement or substrate reduction treatments. Researchers will monitor whether the therapy maintains stable blood counts and is safe over one year.
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Genom att skicka in godkänner du våra Användarvillkor
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Genom att skicka in godkänner du våra Användarvillkor
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Conditions
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