Targeted therapies and holistic interventions, including integrated pharmacy care, are essential to improve psychosocial adjustment and overall well-being in patients with Duchenne Muscular Dystrophy.
Duchenne Muscular Dystrophy (DMD) is a debilitating X-linked genetic disorder characterized by progressive muscle degeneration due to dystrophin deficiency, predominantly affecting males with an incidence of 1 in 3,500 live births. This qualitative review synthesizes literature and patient perspectives to explore the profound impact of DMD on daily life, encompassing functional decline, diminished quality of life (QoL), and psychosocial challenges. Through a pharmacy-focused lens, it examines therapeutic interventions—such as corticosteroids, exon-skipping drugs (e.g., eteplirsen), and emerging gene therapies—and their effects on symptom management, adherence, and long-term outcomes. Key findings highlight relentless physical deterioration leading to loss of ambulation, respiratory complications, and cardiac issues, often compounded by emotional distress, social isolation, and family burden. Pharmacotherapeutic strategies offer hope by mitigating progression and enhancing functionality, yet barriers like side effects, high costs, and access disparities persist. Drawing from clinical studies and lived experiences, this review underscores the need for integrated pharmacy care, including patient counselling and multidisciplinary support, to improve psychosocial adjustment and overall well-being. Ultimately, while DMD profoundly disrupts patient life, targeted therapies and holistic interventions can foster resilience and dignity.
2Urvashi Trivedi *1Astha Suthar (Wed,) studied this question.