Clinical Immunodeficiency Network

One of the first international clinical trials of gene therapy as a treatment for a rare disease, Wiskott-Aldrich Syndrome, has allowed Genethon – already renowned as a unique center for innovation – to gather expertise and skills from around the world. Teams from Genethon, Hôpital Necker Enfants Malades in Paris, Great Ormond Street Hospital in London and the Children’s Hospital in Boston are working closely together to assess the efficacy in patients of a product that was initially developed by Genethon.