An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of Delandistrogene Moxeparvovec Following Plasmapheresis in Subjects With Duchenne Muscular Dystrophy and Pre-existing Antibodies to AAVrh74.
| Zdroj: | Gene Therapy Weekly; 10/11/2024, p131-131, 1p |
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| Abstrakt: | This document provides information about a clinical trial for a gene therapy treatment called delandistrogene moxeparvovec (also known as SRP-9001 or ELEVIDYS) for individuals with Duchenne muscular dystrophy (DMD). The trial is currently not recruiting participants and aims to enroll 16 male children between the ages of 4 and 8. The treatment involves a single intravenous infusion of delandistrogene moxeparvovec after a plasmapheresis procedure, provided that certain antibody levels are sufficiently low. The trial is being conducted by Sarepta Therapeutics, Inc. in the United States. [Extracted from the article] |
| Databáze: | Supplemental Index |
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