Theme: Clinical Trial: Qualifying

Clinical Trials: A mom on dose escalation studies and gene therapy

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Clinical Trials: A mom on dose escalation studies and gene therapy

In this video, the mother of two boys with Duchenne Muscular Dystrophy talks about putting a child in a dose escalating study. She explains the difficult decision of participating in a Phase 1 gene therapy trial at the lowest dose because currently you cannot be re-dosed. She briefly discusses placebo-controlled trials.