”A milestone in gene therapy: proof of long-term efficacy”
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”.
