Telethon announces commitment to gene therapy for Wiskott-Aldrich syndrome. This is the second stage of a path traced by Telethon to prevent gene therapies, potentially capable of changing the natural history of very serious diseases, from being unavailable to patients.
The Foundation will be supported through a rigorous process of regulatory consultancy and pre-authorisation checks in the application for marketing in Europe. The disease affects almost exclusively males and manifests itself from early childhood with recurrent and relapsing infections.