Our mission is:
To advance biomedical research
Right from its beginning Telethon publicly announced that its objective was to assist the growth of excellent scientific research in Italy.
Commitment to treating muscular dystrophy and other genetic disorders
We fund research into genetic disorders, and hereditary pathologies that affect between 24 to 36 million Europeans (in Europe 25) that are often neglected by public funded research and the pharmaceutical industry. In 20 years of research many important results have been achieved. Above all in the treatment of the fourteen children affected by Ada-Scid, a terrible genetic disorder that affects the immune system.
Our vision for 2015 is:
Make available as therapies the results of excellent research selected and sustained over time
In order to put an end to the neglect and disregard of rare disorders a step at a time, we must continue to fund research of excellence and help it to move as much as possible towards reaching clinical application. Once the validity of a therapy has been demonstrated, it is necessary to complete further steps to make it accessible: the industrialisation, production and distribution of the therapy. This stage requires large monetary investments and specific areas of expertise, especially by an industry that generally does not take rare disorders into consideration.