For the roughly 1 in 3,500 males born with Duchenne muscular dystrophy, the disease's physical toll is inseparable from its financial one — and new systematic evidence quantifies just how wide that gap can be. Understanding the true cost architecture of DMD matters not only for health economists and policymakers, but for clinicians helping families plan care trajectories and for researchers benchmarking the value of emerging gene therapies.

This PROSPERO-registered systematic review pooled cost-of-illness data from 19 publications representing 6,993 patients across 14 countries, converting all figures to 2025 international US dollars for comparability. Direct medical costs showed extraordinary variance, ranging from approximately $2,620 to $209,980 per patient annually — a spread that likely reflects disease stage, country-specific healthcare systems, and access to high-cost pharmacological interventions such as exon-skipping therapies and corticosteroid regimens. Direct nonmedical costs — covering items like home modifications, transportation, and assistive devices — added another $5,670 to $103,800 annually. Indirect productivity losses, primarily borne by parents and caregivers rather than patients themselves, contributed a further $400 to $48,390 per year. The authors note that most included studies carried some risk of bias, assessed via the Newcastle-Ottawa scale.

The wide cost ranges are analytically important: they suggest the economic burden of DMD is not a single figure but a trajectory that escalates sharply as patients lose ambulation and require ventilatory support or complex nursing care. This aligns with natural history data showing step-change increases in care intensity around ages 10–14 and again in the late teens. What the review cannot resolve — largely due to data heterogeneity — is how newer disease-modifying treatments are reshaping the cost curve over time. As exon-skipping agents and, potentially, gene replacement therapies enter broader clinical use, the balance between upfront therapeutic costs and downstream care savings will become a central pharmacoeconomic question. This review establishes a rigorous baseline against which those future cost-offset analyses can be measured, making it a valuable reference point despite its inherent cross-national comparability limitations.