Telethon experience and its efforts towards the registration of new advanced therapies

Article type: Research Article

Authors: Paganin, Carla

Affiliations: Telethon Scientific Office, Theleton Foundation ONLUS, Rome, Italy. E-mail: cpaganin@telethon.it

Abstract: The mission of the Telethon Foundation is to boost research towards a therapy for muscular dystrophies and other genetic disorders, giving priority to those diseases neglected by public or private funding. Telethon operates in the Italian environment and has adopted a transparency policy on how donations are distributed to finance research.

Keywords: Telethon, genetic disorders, ADA-SCID, gene therapy

DOI: 10.3233/PPL-2010-0285

Journal: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 133-135, 2010