After 30 years, Genethon now sees its research growing into treatments.
A first gene therapy drug integrating technologies from pioneer research carried out at Genethon is available in the United States and in France for spinal muscular atrophy, a neuromuscular disease that, in its most severe form, kills children before the age of two.
Several other drug candidates, developed by Genethon either alone or with others, are in clinical trials around the world for rare eye, liver, blood, immune system and muscle diseases. Ten or so others are in development.
To learn more about Genethon, watch our video series recounting the incredible saga of this unique laboratory in the world, created by a patient organization and funded by Téléthon donations.
Episode 1: From the human genome to the first gene therapy succes
Episode 2: a new challenge
Episode 3: Gene therapy multiplies victories