Fondazione Telethon is a third-sector organization that has been working since 1990 to advance scientific research toward curing rare genetic diseases. It does this by supporting and implementing the best possible research: from basic research, which is necessary to understand disease mechanisms and identify new therapies, to clinical research, which involves patients. It is a great collective endeavor that feeds on the support of civil society and supporters and has repeatedly generated concrete impacts on patients' lives.

For the Telethon Foundation's mission to be fully accomplished, however, scientific results, however positive, are not enough: the therapies developed must actually be available to patients. Instead, access to treatment is increasingly compromised, particularly for advanced therapies such as gene therapies. In the face of inevitable abandonment of the field by the pharmaceutical industry, there is a risk that patients will no longer have access to life-saving therapies. 

This is what happened in 2022 with the two gene therapies developed by the San Raffaele Telethon Institute for Gene Therapy in Milan for two severe congenital immunodeficiencies-Ada-Scid and Wiskott-Aldrich syndrome. Children with these diseases have been and are at risk of not being able to benefit from these therapies-an unacceptable situation for the Telethon Foundation, which decided to correct it by taking responsibility for producing and distributing them. A commitment compl