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Investigational gene therapy shows benefits in two boys with DMD
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Investigational gene therapy shows benefits in two boys with DMD
An investigational gene therapy called GNT0004 has demonstrated sustained stabilization of motor function at up to two years of follow-up in two boys with Duchenne muscular dystrophy (DMD). The boys received what was determined to be the effective dose of GNT0004, which was developed by Genethon, during the initial Phase 1/2 of a multiphase clinical trial named GNT-016-MDYF. The new data were presented earlier this month at the annual meeting of…
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