Jenn M

Mother to Austin(20), Max(17), James(11) and Norah(8). Austin & Max have Duchenne Muscular Dystrophy & James has Primary Immune Deficiency

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.