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We've funded more than 2700 projects and 1600 researchers to give people affected by a rare genetic condition a new hope.
What we do
We were made to give people affected by a rare genetic condition an answer – treatment for their condition. Rare genetic conditions are not statistically relevant when considered individually, but the same can’t be said about a person’s life.
We support the best projects chosen using a rigorous and transparent selection process, following consultation with an internationally-renowned committee of qualified scientists.
Orchard Therapeutics Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)
First gene therapy to receive full EU marketing authorization for eligible MLD patients. One-time treatment with Libmeldy has been shown to preserve motor and cognitive function. Achievement shared with research alliance partners Fondazione Telethon and Ospedale San Raffaele.
A novel finding on Kabuki syndrome, a rare genetic disease
The research, coordinated by the University of Trento, has recreated for the first time the pathological condition in a test tube discovering what happens in the cell nucleus and what hinders the formation of cartilages and bones.
Awarded Telethon Farmindustria Prize to three young researchers
Farmindustria and Fondazione Telethon have awarded three prizes of 10,000 euros to young researchers who work in the neonatology field.
How we fund research?
We select the best research projects using an internationally-recognised selection method i.e. the peer review process.
Millions of euros invested