We were made to give people affected by a rare genetic condition an answer - treatment for their condition.
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, but the same can't be said about a person's life.
What is our response to this silent emergency?
The journey of scientific research towards a cure is lengthy, demanding, and punctuated by obstacles and successes, involving small steps towards enormous goals.
We develop innovative treatments that are already saving the lives of children such as Ana Carolina
We provide a diagnosis for patients such as Nina suffering from extremely rare and unknown conditions
We work continually to improve the lives of patients such as Michele with neuromuscular conditions
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We work to transform the results of excellent, supported scientific research into drugs and treatments available to patients all over the world.
For some years now this vision has been a reality, thanks to innovative treatments that attract the attention of increasing numbers of pharmaceutical groups, and partnerships with public and private health facilities, so our work doesn't just stop at successful scientific results, but actually becomes a reality for each and every patient.
We pursue our vision in two ways:
- by continually supporting our Milan and Pozzuoli institutes, which are centres of excellence attracting researchers from all over the world.
- by funding the best research in Italy, selecting the most promising projects using transparent methods that reward merit and excellence.