Emerging Therapies in Duchenne Muscular Dystrophy: A Comparative Analysis of Gene Therapy and Pharmacological Approaches

Abstract

Duchenne muscular dystrophy (DMD) is a progressive and severe X-linked recessive disorder characterized by progressive muscle weakness due to degeneration of muscle fibers resulting in a shortened life span. For management, there are pharmacological therapies including corticosteroids, exon-skipping drugs, stop codon readthrough drugs and utrophin modulators. There is also the possibility of gene therapy through gene replacement therapy and CRISPR-Cas9 gene editing. This thesis compares these treatment strategies with the understanding that through continued research, better patient outcomes can be achieved for such a devastating disease.