Since 1990, Fondazione Telethon has been at the forefront of transforming scientific research into real solutions for people living with rare genetic diseases. It funds excellence in research, from understanding disease mechanisms to developing innovative therapies and conducting clinical trials with patients. This is a major collective project that, thanks to the support of millions of people, has already changed the lives of many children and their families.

However, the mission does not stop at scientific discovery: a therapy is truly such only when it is accessible to those who need it. For this reason, Fondazione Telethon works to ensure that research results become concrete treatments that remain available over time.

At a time when the pharmaceutical industry is increasingly withdrawing from the rare disease sector, there is a real risk that life-saving therapies—even those already developed—may not be made available to patients. To ensure continuity for two gene therapies for severe pediatric immunodeficiencies (ADA-SCID and Wiskott-Aldrich syndrome), developed at the San Raffaele Telethon Institute in Milan, the Foundation has therefore chosen to take direct responsibility for their production and distribution.

A complex but necessary commitment, to guarantee patients a tangible hope.

Learn more: https://www.fondazionetelethon.it/