We work everyday for:

Every life counts

Mostly overlooked by large public and industrial investments, orphans of research and drugs: rare genetic diseases taken individually are not statistically significant. But can we say the same for somebody’s life?

Why do we study genetic diseases?

Research can give real hope

By funding research we can provide answers and therapies to people who otherwise, in many cases, would not have any hope.

Learn more about our mission.

Latest news

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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...
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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 ...
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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...
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GSK receives positive CHMP opinion in Europe for StrimvelisTM, the first gene therapy to treat very rare disease, ADA-SCID
GlaxoSmithKline (LSE/NYSE: GSK) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), in conjunction with the Committee for Advanced Therapies (CAT), has issued a positive opinion recommending marketing authorisation for Strimvelis ...