Telethon Foundation is seeking approval for a gene therapy to treat the rare disease Wiskott-Aldrich syndrome.

Fondazione Telethon, an Italian biomedical charity focused on rare genetic diseases, has submitted a Marketing Authorization ...

Thirty patients have received the gene therapy within a clinical trial and early-access program, and the foundation also hopes to seek approval in the US.

Italian research charity Telethon Foundation is one step closer to getting a gene therapy to treat rare disease Wiskott-Aldrich syndrome WAS approved in the European Union. The gene therapy ...