Quantifying the Hidden Cost Burden of Duchenne Muscular Dystrophy on U.S. Households

For families caring for individuals with Duchenne muscular dystrophy (DMD), the disease’s toll extends far beyond the clinic. A recent caregiver survey conducted by Innis et al. highlights a sobering reality: households often shoulder tens of thousands of dollars in un-reimbursed, out-of-pocket expenses—costs that escalate with disease progression and frequently go unrecognized by healthcare systems.

A National Snapshot of Financial Strain

This cross-sectional study surveyed 90 U.S.-based caregivers of individuals with DMD who had incurred at least one home or vehicle-related expense in the past five years. Their responses, representing 106 individuals with DMD, offer granular insights into how families adapt homes, purchase specialized equipment, and cover recurring non-reimbursed healthcare costs.

Key findings include:

• Average home and vehicle-related expenses totaling $78,303 over five years, with 64% of households reporting costs for handicap-accessible vehicles alone (mean conditional cost: ~$48,000).
• Medical equipment expenses adding another $14,071, driven primarily by powered wheelchairs and assistive respiratory devices.
• Non-reimbursed healthcare services and prescription drugs accounting for over $13,600 annually, with wide variance driven by caregiving needs and disease stage.

Insurance gaps loom large. The data also show that caregivers covered 76.4% of home and vehicle-related expenses directly from household income. Government programs and charitable support made modest contributions, averaging just 5.4% and 18.2%, respectively. A similar pattern held for medical devices and health services, underscoring the systemic lack of financial safety nets for DMD-related functional needs.

Additionally, stratified analyses confirmed what many clinicians and families already suspect: as physical function declines, expenses rise sharply. Compared to ambulatory individuals, households caring for those in non-ambulatory stages faced nearly three times higher home and vehicle-related costs (mean: $87,168 vs. $28,898). Medical equipment costs and in-home professional caregiving similarly tracked with disease progression and upper-limb impairment.

Notably, some of the most expensive accommodations—such as installing elevators or modifying kitchens—were often forgone due to financial barriers, even when clearly needed.

Policy Implications: Toward Holistic Coverage Models

The findings raise important questions for policy and payer systems. While clinical trials and gene therapies garner attention and investment, the everyday costs of living with DMD remain largely unaccounted for in standard reimbursement frameworks. The authors suggest that more comprehensive coverage policies—including funding for non-medical but medically necessary home and vehicle adaptations—are essential for equity in DMD care.

Moreover, this study reinforces the need for multidimensional burden assessments in rare diseases. Beyond direct medical costs, long-term quality of life, caregiver capacity, and financial solvency are all deeply influenced by out-of-pocket expenditures that often go untracked.

Reference:

Innis B, Jarvis J, Renteria T, Audhya I. Household costs in the United States for accommodating functional impairments associated with Duchenne muscular dystrophy: results from a caregiver survey. Orphanet J Rare Dis. 2025;20:301. doi:10.1186/s13023-025-03794-1.