DMD Care Expands With Multidisciplinary Management, Earlier Pathology Focus
Duchenne muscular dystrophy care is shifting toward broader multidisciplinary management and closer attention to early pathology, biomarkers, and treatment timing. Clinicians described an expanding treatment landscape and identified neurology, cardiology, pulmonary, and endocrinology as core priorities.
Duchenne muscular dystrophy care has undergone a dramatic transformation over the past five to ten years, with clinicians now navigating an expanding and evolving treatment landscape. Discussions during Duchenne Muscular Dystrophy Awareness Week highlighted essential multidisciplinary teams, family-centered visits, and growing focus on early pathology, biomarkers, treatment timing, and combination strategies as gene-targeted therapies expand.
Clinicians described the neurologist as the "captain of the ship," coordinating care across a wide range of subspecialties. Key specialists include cardiologists, pulmonologists, endocrinologists, physical and occupational therapists, orthopedics, palliative care, and nutrition. Care must evolve with disease stage: in early ambulatory phases, physical and occupational therapy take priority, while cardiac, pulmonary, and endocrine needs become prominent as the disease progresses.
The discussion also addressed the real-world challenge of not every institution having access to comprehensive interdisciplinary teams. Neurology, cardiology, pulmonary, and endocrinology were identified as non-negotiable priorities. Participants also emphasized asking families what concerns they want to address at each visit, the critical but often overlooked role of psychiatry and psychology, and the need to meet families where they are culturally and linguistically.
The evolving landscape of DMD biology and therapy included evidence that disease processes begin earlier than once recognized and the growing importance of immunologic factors in shaping progression and therapeutic response. The conversation explored how neuromuscular specialists should approach treatment timing and combination strategies, the evolving interpretation and limitations of biomarkers such as creatine kinase and dystrophin expression, and what emerging gene therapy platforms may signal for care heading into 2026 and beyond.
A forthcoming discussion in the series will provide a comprehensive overview of mutation-specific and non-mutation-specific DMD therapies available in clinical practice, including corticosteroids, exon skipping, and gene therapy.