The Telethon Foundation lands in the United States with a new challenge that confirms the excellence of research made in Italy. For the first time in the world, a non-profit organisation has received authorisation to distribute a therapy resulting from its own research and development in the United States.
The US Food and Drug Administration (FDA) has approved an ex vivo gene therapy (Waskyra) for patients suffering from Wiskott-Aldrich syndrome (Was), a rare and severe genetic immunodeficiency. The Fda's approval follows the positive opinion of the European Medicines Agency's (EMA) Chmp for the same therapy, which came a few weeks ago, and confirms the effectiveness of a model that has become unique worldwide.
