Dmd – animal models

Duchenne muscular dystrophy (DMD) is an inherited and lethal disease leading to the lack of expression of dystrophin and muscle degeneration for which there is no curative treatment. Current life expectancy of DMD patients is 20 to 40 years with final cardio-respiratory complications. Clinical trials are ongoing using rAAV-microdystrophin (rAAV-MD) gene therapy. The aim being a shift from DMD toward a milder dystrophinopathy, the Becker muscular dystrophy (BMD). However, BMD patients still exhibit muscle decline and die before the age of 60.

Source: Neuromuscular Disorders - September 19, 2021 Category: Neurology Authors: A. Creism éas, C. Gazaille, A. Bourdon, A. Lafoux, M. Allais, V. Le Razavet, M. Ledevin, T. Larcher, G. Toumanianz, I. Anegon, O. Adjali, C. Huchet, C. Le Guiner, B. Fraysse Source Type: research