Therapeutic approaches for Duchenne muscular dystrophy: old and new
SJ Mackenzie, S Nicolau, AM Connolly… - Seminars in Pediatric …, 2021 - Elsevier
Duchenne muscular dystrophy (DMD) is marked by pathogenic variants in the DMD gene,
leading to reduced or absent dystrophin translation, muscle fiber destruction, loss of
ambulation, cardiomyopathy, respiratory failure, and eventually death. Disease progression
is slowed with use of prednisone or other corticosteroid agents. Gene replacement therapy,
which is one of the focus points of this review, has emerged as the most promising potential
treatment for DMD, though alternative RNA-based strategies have been employed for …
leading to reduced or absent dystrophin translation, muscle fiber destruction, loss of
ambulation, cardiomyopathy, respiratory failure, and eventually death. Disease progression
is slowed with use of prednisone or other corticosteroid agents. Gene replacement therapy,
which is one of the focus points of this review, has emerged as the most promising potential
treatment for DMD, though alternative RNA-based strategies have been employed for …