We work everyday for:

Every life counts

Mostly overlooked by large public and industrial investments, orphans of research and drugs: rare genetic diseases taken individually are not statistically significant. But can we say the same for somebody’s life?

Why do we study genetic diseases?

Research can give real hope

By funding research we can provide answers and therapies to people who otherwise, in many cases, would not have any hope.

Learn more about our mission.

Latest news

GSK receives positive CHMP opinion in Europe for StrimvelisTM, the first gene therapy to treat very rare disease, ADA-SCID
GlaxoSmithKline (LSE/NYSE: GSK) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), in conjunction with the Committee for Advanced Therapies (CAT), has issued a positive opinion recommending marketing authorisation for Strimvelis ...
Holger Lerche, University of Tübingen (Germany)
Holger Lerche received his education in 1988-1994 (Medical Studies at the University of Munich) and in 1995-2006 at the University of Ulm.He is currently a Full Professor of Neurology, Clinical Director and Head of the Dept. of Neurology and Epileptology at the University of Tübingen.Dr. Lerche’...
Steven Wilson, University College London (UK)
Steven Wilson joined the Scientific and Medical Committee of Telethon in 2013.Stephen W. Wilson received his BSc in Biological Sciences from University of Leicester in 1984 and his PhD in Neurobiology from the University of London in 1988. From 2002 he is Professor of Developmental Genetics at t...
Frances Platt, University of Oxford (UK)
Frances Platt joined the Scientific and Medical Committee of Telethon in 2015.In 1982, Frances Platt graduated in Zoology at Imperial College, London. She then obtained a research doctorate in Animal Physiology at the University of Bath in 1985. Between 1986 and 1987 she held a post-doc at Washin...