Therapeutic Approaches for Duchenne Muscular Dystrophy: Old and New.

ABSTRACT

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 patients with specific pathogenic variants. While challenges remain, many of these novel therapeutic approaches hold promise for treating this devastating disease.