Pharmacotherapy of Duchenne Muscular Dystrophy

Abstract

Duchenne Muscular Dystrophy (DMD) is an X-linked recessive monogenic disease characterized by a rapid lethal course featured by progressive muscle atrophy and weakness. In this paper, I review the new innovative approaches that have been recently developed, which are aiming at treating DMD by restoring the expression of the deficient dystrophin using gene replacement therapy, exon skipping therapy, genome editing, and stop codon read-through agents among other strategies. Drawbacks, advantages, and feasibility of incorporation in DMD treatment will be discussed.