We are a non-profit organisation recognised by the Italian Ministry of Education, Universities and Research. We were founded in 1990 in response to calls by people suffering from rare diseases.
Right from the start, we have always listened to patients, to provide the answers they need. Every day.
We manage fund raising activities and guarantee the necessary resources for our research projects. These projects are subjected to rigorous assessments on the part of an independent, international Scientific and Medical Committee.
Our Board of Directors
- Simona Agnes
- Alessandra Colonna
- Davide Dattoli
- Alberto Fontana
- Giovanni Malagò
- Giovanni Manfredi
- Mariangela Marseglia
- Luca di Montezemolo (Presidente)
- Francesca Pasinelli (Consigliere Delegato)
- Michele Pontecorvo
- Lupo Rattazzi
- Omero Toso (Vice presidente)
How we fund research
Research funds are assigned throught the help of:
Scientific and Medical Committee: follows the peer-review procedure. Before the plenary discussion, each proposed project is examined by three committee members and at least two external reviewers selected from the international community. Thanks to this process, we can be sure that only the best projects are chosen.
Scientific Steering Committee: the task of the Scientific Steering Committee is to support the overall policy and management decisions of the Board of Directors within the framework of biomedical research. The Scientific Steering Committee composition is the following:
- Virginie Bros-Facer, EURORDIS-Rare Diseases Europe (Paris, France)
- Bev Holmes, Michael Smith Foundation for Health Research (Vancouver, Canada)
- Naomi Taylor, Institute of Molecular Genetics of Montpellier (Montpellier, France) and National Human Genome Research Institute, NIH (Bethesda, MD - USA)
- Leonard Bell, Alexion Pharmaceuticals (Boston, MA - USA)
- Michael Caplan, Department of Cellular and Molecular Physiology, Yale University School of Medicine (New Heaven, CT - USA)
- Alexandre Mejat, AFM-Téléthon (France)
- Olaf Riess, Institute of Medical Genetics and Applied Genomics, Medical Faculty and University Hospital, Eberhard Karls University, (Tübingen, Germany).
Finally, we operate on the basis of a quality, ethical and supportive management and administration system. More about the Code of Ethics and Model 231 here.
On 15th May 2023 has been submitted to EMA the request for the MAH (Marketing Authorization Holder) transfer from Orchard to Fondazione Telethon ETS, which will lead for the first time the Organization to manage a drug on the market, Strimvelis.
Therefore, to cope with the aforementioned task, Fondazione Telethon ETS has appointed:
- Luisa Angela Gioeni as Head of Pharmacovigilance, according to Article n. 219 of Legislative Decree of 24th April 2006 and to the Implementing Regulation (EU) n. 520/2012 of the 19th June 2012 Commission (Email: [email protected]);
- Alessandra Schiavo as Head of Scientific Service, according to Article n. 219 of Legislative Decree of 24th April 2006 (Email: [email protected])
