[Comment] Delandistrogene Moxeparvovec gene therapy for Duchenne muscular dystrophy: the way forward in South Asian countries

With growing use of gene therapies for rare diseases in clinical practice, it becomes important to analyse utility and challenges of the recently-approved gene therapy for Duchenne muscular dystrophy (DMD) in South Asian context. DMD is a rare, X-linked severe childhood muscular dystrophy caused by pathogenic variations (mostly deletions) in dystrophin gene. It leads to progressive weakness with loss of ambulation in adolescence culminating in early mortality.1 Despite several advancements over the last few decades, there is a significant unmet need for a curative therapy for DMD (Fig.