A great result
For all children with ADA-SCID, a rare immunodeficiency, gene therapy is finally available; thanks to an agreement signed in 2010 with GlaxoSmithKline, the outstanding results obtained by researchers from the San Raffaele Telethon Institute for Gene Therapy in Milan have been turned into a treatment accessible to all patients in Europe.Read more
Strimvelis, new hope
The European Commission has given the green light for the use of gene therapy for ADA-SCID as a drug: see the comments by some of the main protagonists of this huge success.
Gene therapy stories
THE DISEASES THAT OUR RESEARCH INSTITUTES INVESTIGATE
The research carried out at our institutes has pinpointed promising therapeutic strategies for combatting a number of diseases. Such results require years of investigation of the mechanisms underlying genetic diseases, and for development of innovative forms of treatment. Concrete answers are now emerging. For all patients.