<p>Duchenne muscular dystrophy (DMD) is an X-linked disorder affecting ~1 in 3,600 male births, with an estimated 400,000–500,000 affected children in India. Mutations in the <i>DMD</i> gene cause dystrophin deficiency, leading to progressive muscle degeneration and fibrosis. Early diagnosis through multiplex polymerase chain reaction (mPCR), MLPA (multiplex ligation-dependent probe amplification), or next-generation sequencing (NGS) enables timely interventions, including physiotherapy, cardiac and pulmonary care, and genetic counselling. Advanced therapies target dystrophin restoration: exon skipping (eteplirsen, golodirsen, casimersen), stop codon readthrough, and gene therapy. In 2023, the US FDA approved delandistrogene moxeparvovec-rokl (Elevidys), an AAV-delivered microdystrophin gene therapy. Agents such as vamorolone and givinostat address secondary pathology by reducing inflammation, fibrosis, and muscle degeneration. However, several limitations persist: the mutation-specific nature of many therapies restricts their applicability to a small subset of patients; high costs significantly limit access, especially in resource-limited settings like India; the risk of insertional mutagenesis with gene therapy raises safety concerns; and critically, the long-term safety and clinically meaningful benefits of these novel treatments remain largely unknown. Although clinical trials and indigenous gene therapy efforts are underway in India, widespread use is still constrained by financial, infrastructural, and awareness-related challenges. Improving DMD care in India needs awareness campaigns, advocacy for rare disease policy, and global collaboration. Strengthening NGS-based genotyping, natural history studies, and expanded carrier, prenatal, and neonatal screening can reduce disease burden and enhance clinical trial readiness. In particular, reference to counseling and prenatal testing, which is the most cost-effective and widely utilized form of secondary prevention, is critical in the Indian context.</p>