Frédéric Revah, CEO of Genethon discusses Genethon’s major contribution to therapeutic innovation
“Recent data presented at the Muscular Dystrophy Association Clinical and Scientific Conference, in March, have detailed long-term benefit of gene therapy in infants affected with Spinal Muscular Atrophy type 1 (Mendell at al.; Connolly et al.).
Virtually all patients affected with this deadly disease die before the age of two if untreated. Strikingly, more than seven years after treatment with Zolgensma® all patients treated at
therapeutic dose were alive and had maintained clinical benefits observed in initial reports”.