Gaps in Care for Childhood SMA

EP: 1.Advancements & Challenges in Muscular Dystrophy Treatment

EP: 2.Data on Duchenne Muscular Dystrophy Treatment From MDA 2025

EP: 3.Key Takeaways From DMD Treatment Data

EP: 4.Overview of Spinal Muscular Atrophy

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EP: 5.Gaps in Care for Childhood SMA

EP: 6.The Impact of Intrathecal Onasemnogene Abeparvovec Therapy in SMA

Gaps in Care for Childhood SMA

Despite the development of highly effective treatments for spinal muscular atrophy (SMA), significant gaps in care persist that limit equitable access to these life-changing therapies. SMA has emerged as a potential model for treating genetic diseases, with treatments capable of providing patients with nearly normal lives and function. However, the success stories primarily come from developed countries, where research infrastructure and health care systems are well established. The high cost of newly developed medications, combined with the need for sophisticated medical infrastructure, creates substantial barriers to global delivery of SMA care, highlighting a concerning disparity in treatment access.

Racial and ethnic disparities are readily apparent in SMA treatment data, despite ongoing efforts to ensure equitable patient recruitment in clinical trials. These disparities reflect broader systemic challenges in health care delivery, where advanced genetic therapies remain concentrated in developed nations with robust health care infrastructure. The life-altering potential of these therapies makes the access gap particularly concerning, as they can dramatically improve patient outcomes and activities of daily living. The contrast between the therapeutic potential and limited global accessibility underscores the urgent need for targeted interventions to address these inequities.

Addressing SMA care gaps requires multifaceted approaches that tackle both economic and infrastructure barriers. While medication costs may change over time, immediate solutions must focus on understanding specific regional infrastructures and developing creative approaches to overcome existing barriers. Future research should prioritize diversity in clinical trials, including patients with varying performance statuses to improve accessibility and impact outcomes across broader populations. Creative solutions that address regional differences in health care infrastructure, combined with efforts to reduce economic barriers, will be essential for ensuring that the remarkable advances in SMA treatment can benefit patients worldwide rather than remaining concentrated in developed countries with existing health care advantages.