Background
Despite some progress in global health, childhood disorders remain the leading cause of disease burden, having the slowest decline in disability-adjusted life years (DALY). The advent of novel disease-modifying therapeutics in spinal muscular atrophies (SMAs) has turned this potentially fatal disease into a treatable one, and many patients may live close to a normal life. These new therapies bring promise for SMA patients, but they are expensive, are not available across the world, and can potentially widen the gaps in healthcare disparities in SMA patients.
Objectives
It is critical to identify and address sources of such health inequalities and to plan to address them. By studying the gaps in SMA care and providing recommendations, we aim to include strategies that can be applicable and can target disparities affecting a broad range of neuromuscular disorders.
Results
In this work, we review such gaps, including newborn screening availability, medication accessibility, medication costs, distribution problems, and health insurance access. We also discuss the challenges in clinical access and follow-up, the lack of racial diversity, and age-dependent barriers in SMA research and healthcare access. Finally, we propose strategies for the provision of the standard of care for SMA patients. These include strategies that promote newborn screening, health policy, and advocacy approaches, as well as recommendations for clinical continuity. We also emphasize the role of research in further clarifying and addressing sources of inequality and care.
Conclusions
Childhood health and well-being are crucial, and it is paramount that gaps in their care are addressed swiftly in a rapidly changing healthcare environment. Therefore, ensuring equitable healthcare access to disease screening and lifesaving medications is not just a recommendation but a call to action that can promote health for all.