If you believe in what we do, all we ask of you that you find out more. You’ll discover what we can do together, and what more we can do together!

Our official positions

Our commitment to providing assistance to patients suffering from rare genetic diseases means we must assume clear, unambiguous positions in regard to the manner in which research is conducted in Italy and the means required to remove obstacles to achieving our final goal, namely treatments.

READ ON

Latest news

immagine-anteprima-news
Editas Medicine Announces Scientific Multi-Year Collaboration with Fondazione Telethon and Ospedale San Raffaele
Editas Medicine, Inc. (NASDAQ: EDIT), a leading genome editing company, and Fondazione Telethon and Ospedale San Raffaele, which operate a joint research collaboration known as the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) have entered into a scientific collaboration to research...
immagine-anteprima-news
Preliminary results reporting the safety and effectiveness of gene therapy in patients with metachromatic leukodystrophy published in The Lancet
The gene therapy developed in the laboratories of the San Raffaele Telethon Institute for Gene Therapy in Milan (SR-Tiget) remains effective as a potential early treatment of metachromatic leukodystrophy (MLD), a rare neurodegenerative disease caused by the alteration of a gene called arylsulfat...
immagine-anteprima-news
Strimvelis receives European marketing authorisation to treat very rare disease, ADA-SCID
GlaxoSmithKline (GSK), Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR) today announced that the European Commission has approved Strimvelis, the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to ...
immagine-anteprima-news
GSK, Fondazione Telethon and Ospedale San Raffaele announce publication of pivotal safety and efficacy of gene therapy for children with ADA-SCID
GSK, Fondazione Telethon and Ospedale San Raffaele today announced the publication in BLOOD of the long-term safety and efficacy data from an analysis of 18 children with ADA-SCID treated with hematopoietic stem cell gene therapy between 2000 and 2010 at the San Raffaele Telethon Institute for G...