Duchenne Muscular Dystrophy: Clinical Innovations, Ethical Considerations, and Evolving the Path to Adulthood

Duchenne Muscular Dystrophy (DMD) is a progressive and life-limiting neuromuscular disorder with profound implications for affected individuals and their families. Advances in genetic and molecular therapies, including exon skipping agents, gene therapy, and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-based approaches have ushered in a new era of disease-modifying treatments. However, these breakthroughs also introduce complex ethical and psychosocial challenges. As we continue to extend life expectancy and improve motor outcomes for individuals with DMD, there is also a need to ensure autonomy-supportive environments, particularly during the transition to adulthood. This review provides a comprehensive exploration of the evolving landscape of DMD management, the long-term and ethical considerations of experimental therapies, and the psychosocial impacts on patients and caregivers. In the context of recent major advancements in medical care, we reflect on the impact of the growing group of DMD "boys" navigating the transition to adulthood. Lastly, this review highlights the critical need for multidisciplinary care models that address not only the biological but also the evolving psychological and social dimensions of the disease. By synthesizing perspectives from treatment innovation and ethical discourse, this article equips readers with a holistic understanding of the transformative potential and challenges associated with DMD therapies. This review aims to support multidisciplinary neuromuscular clinics to more fully meet the needs of an increasingly diverse population of patients with an ever-expanding potential.