
What we do
We advance excellence and innovation in rare genetic research, supporting outstanding scientists and breakthrough ideas. Through our institutes and funding programs, we drive progress from discovery to therapy, ensuring that pioneering science translates into real solutions and lasting hope for patients.

Research generates hope
They have awful names and in some cases have no name at all, because they are so rare or unknown. They are rare and affect a small number of people, most frequently appearing in the first few years of life and changing the destiny of that child. We fight them because every life counts.
How we use the funds collected
Thanks to support from millions of Italians, we fund the best scientific research into rare genetic conditions.
- 741 millions of euros invested
- 1.916 Researchers
- 3.118 projects funded

The legacy we share with our supporters, an investment for the future of everyone.
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Diagnosis and Treatment
We have developed cutting-edge treatment models and are giving a name to unknown conditions.
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Supporting research and patients
We work with researchers and patients to support them in the long journey towards a cure.
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Research management
Rigorous, transparent methods enable results to be achieved effectively and efficiently.
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