Unmet Needs in the Care of Patients with Duchenne Muscular Dystrophy in Brazil

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Abstract
Duchenne muscular dystrophy is a rare, progressive neuromuscular disorder primarily affecting boys, and it follows a predictable course. Early intervention is essential for effective management, but disparities in the care of patients with rare diseases hinder access to optimal treatment. To identify unmet needs and challenges in the care of patients with Duchenne muscular dystrophy within the Brazilian public health system compared with the private system. A cross-sectional observational study using the Delphi method was conducted with ten neurologists specialized in Duchenne muscular dystrophy. The specialists participated in rounds of surveys to reach consensus on key issues, including diagnosis, treatment, and care. Data was analyzed using descriptive statistics. According to the Delphi panel, the public health system had an average diagnostic delay of 25 months compared with 10 months in the private sector. Although genetic testing is critical, it is not funded by the public health system. Other barriers included delayed corticosteroid treatment, limited access to multidisciplinary care, and insufficient medical devices. Patients in the public health system lost ambulation earlier (11–12 years of age) than those in the private sector (13–14 years of age). Life expectancy was significantly shorter in the public system, averaging 19 to 20 years compared with 26 to 27 years of age in the private sector. There are significant disparities in the care of patients with Duchenne muscular dystrophy within Brazil's public health system, resulting in worse outcomes. Enhancing access to genetic testing and early multidisciplinary care is crucial to improve the quality of life and survival of these patients.

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