Fondazione Telethon is an Italian non-profit organization committed to advancing research on rare and complex genetic diseasesFor over 35 years, we have supported high-impact science aimed at developing innovative treatments and improving the lives of people affected by these conditions. 

We develop innovative treatments that are already saving the lives of children such as Ana Carolina

Often neglected by large public or industrial investment schemes, and the poor relation when it comes to research and drug treatments - rare genetic conditions are not statistically relevant when considered individually.  

3,5%-5,9% of the world population affected
6-8.000 known rare conditions
2.1-3.500.000 people affected in Italy

What is our response to this silent emergency?

We aim to: 

  • develop innovative treatments and therapies, aiming to translate research into real-world health benefits. 
  • fund cutting-edge research on genetic diseases, with a focus on rare and underserved conditions. 
  • help shape regulatory frameworks that ensure high standards and sustainability in the development of gene and cell therapies. 
  • raise awareness about the burden of genetic diseases and the importance of sustained scientific and societal commitment. 

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