Advancements and challenges in muscular dystrophy: A comprehensive review of current research and therapeutic approaches

Agnimandala, Vasundhara and Ayesha, Shaik Beebi and Dhiraja, Ankusarao Satya and Biradavolu, Vyshnavi and Kantamaneni, Padmalatha (2025) Advancements and challenges in muscular dystrophy: A comprehensive review of current research and therapeutic approaches. World Journal of Biology Pharmacy and Health Sciences, 21 (3). pp. 307-318. ISSN 2582-5542

Muscular Dystrophy (MD) refers to a group of hereditary disorders that are characterized by progressive muscle weakness and degeneration, with Duchenne Muscular Dystrophy (DMD) being the most common and severe form. Despite significant advancements in research and therapeutic strategies, MD remains an incurable condition, presenting substantial challenges for both patients and clinicians. This comprehensive review aims to provide an in-depth overview of the current state of MD research, highlighting recent advancements in genetic therapies, pharmacological interventions, and cell-based treatments. We also examine the challenges associated with these therapeutic approaches, including issues of efficacy, safety, and accessibility. Key areas of progress include gene-editing technologies, such as exon-skipping and CRISPR-based therapies, which have shown promise in restoring dystrophin expression in muscle tissue. Additionally, corticosteroids remain a cornerstone in managing disease progression, although there are concerns regarding their long-term side effects. Stem cell therapies and novel small-molecule drugs also hold the potential for muscle regeneration and symptom relief, although these strategies are still undergoing clinical evaluation. Furthermore, the review discusses the complexities of the clinical trial design, patient stratification, and the necessity for personalized medicine approaches to optimize treatment outcomes. Finally, we address the socio-economic and ethical challenges accompanying new treatments, emphasizing the need for a collaborative, multidisciplinary effort to implement these advancements in clinical practice. This article seeks to provide a comprehensive understanding of the landscape of muscular dystrophy research and therapy, offering insights into promising avenues besides the obstacles that remain in the pursuit of effective treatments.