Duchenne muscular dystrophy: Current state and therapeutic perspectives

Duchenne muscular dystrophy is one of the most frequent muscular dystrophies, with 250,000 affected people worldwide. It results from mutations occurring in the dystrophin gene, the largest known in the human genome. Despite current therapies allow for a better clinical prognosis, the life span and the quality of life of patients still remain clearly lower than those in the general population. New therapeutic approaches are currently investigated, giving new perspectives and hopes for the care of this severe disease. (C) 2021 l'Academie nationale de medecine. Published by Elsevier Masson SAS. All rights reserved.