To understand caregiver perspectives on Duchenne Muscular Dystrophy (DMD) care, including compliance to care guidelines and barriers to care.

DMD is a severe, progressive neuromuscular disorder caused by dystrophin gene mutations, primarily affecting males. Although care guidelines promote multidisciplinary management, studies have shown inconsistent and often suboptimal compliance across the U.S.

Caregivers (≥ 18 years) of individuals with DMD were recruited via Albany Medical Center and national patient advocacy organizations. Participants completed an online survey assessing compliance with 2018 care considerations. Thematic analyses were performed for open-ended data, and descriptive analyses for close-ended data. For individuals aged ≥ 6 years, compliance scores were calculated based on the number of care recommendations met.

A total of 146 eligible caregivers completed the survey. The mean age of individuals with DMD was 16.6 ± 8.26 years (range 2–50), with 59% non-ambulatory (n = 86). The mean compliance score was 9.8 ± 2.7 (69.6% ± 19.5 of 14 care recommendations). Compliance scores were significantly higher in children than adults (p < 0.001), clinical trial participants than non-participants (p = 0.018), individuals diagnosed before age 1 than between ages 5-7 (p = 0.003), and individuals from households with another disability than those without (p = 0.025). While most caregivers were satisfied with their child’s overall health care, neuromuscular follow-up, involvement in decision-making, and insurance, they also expressed a need for tools such as checklists and educational brochures to navigate care. Caregivers’ top priorities for improvement included their child’s neuromuscular, cardiology, and psychosocial care. Thematic analyses identified the most common barriers to care: cost, limited provider availability, and inadequate support for adults with DMD.

Despite general satisfaction and compliance with many aspects of care, notable gaps persist. These findings underscore the importance of caregiver-informed strategies to strengthen adherence to guidelines and improve multidisciplinary DMD management.