Duchenne muscular dystrophy (DMD) is a rare progressive muscular disease that primarily affects boys. A lack of comprehensive care for patients living with DMD is directly associated with a compromised quality of life for those affected and their caregivers. This disease also has a huge economic impact on families as its treatment requires substantial direct, indirect, and informal care costs.
This study presents a protocol developed to evaluate the feasibility and efficacy of a patient-centered care model for children with DMD. The care model is designed with the aim to empower families, improve quality of life, and reduce economic burden on their families.
This report outlines a research proposal developed to study the impact of a patient-centered care model for patients with DMD in low- and middle-income countries (LMICs). This study is expected to provide evidence of whether a multicomponent, patient-centric intervention could reduce economic burdens on families and improve their quality of life. The results of this study could guide policy makers and health professionals in low- and middle-income countries to facilitate a comprehensive care program for patients living with DMD. The economic impact of a rare disease is an important consideration to formulate or evaluate any health policy or intervention related to new treatments and financial support scheme. Read the full article here.