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Getting the latest healthcare news for you
Getting the latest healthcare news for you
Spinal muscular atrophy (SMA) care has been revolutionized by disease-modifying therapies and newborn screening, enabling children to hit motor milestones once thought impossible. But experts stress that DMTs alone aren't enough — comprehensive, lifelong multidisciplinary care covering mobility, breathing, nutrition, and more remains essential. The next frontier is improving outcomes for patients who start treatment late or have lived with SMA for years.
The SMA treatment landscape has undergone a dramatic transformation over the past decade. Disease-modifying therapies (DMTs) like nusinersen, risdiplam, and onasemnogene abeparvovec — combined with the addition of SMA to newborn screening panels — have shifted care from managing decline to actively changing the disease course. Children are now reaching motor milestones that were once considered out of reach, and the focus has expanded well beyond survival.
But experts are quick to note: more progress doesn't mean simpler care. As patients live longer, clinicians are managing a broader and more complex set of needs — from respiratory health and orthopedic issues to nutrition, fatigue, and the challenging transition from pediatric to adult care. Real-time communication between specialists, dedicated care coordinators, and bundled clinic visits are emerging as critical tools for keeping multidisciplinary teams functioning effectively. Adult care infrastructure, in particular, is still catching up to the growing population of SMA patients aging out of pediatric programs.
Key Takeaways:
Why it matters: SMA is no longer the uniformly devastating diagnosis it once was — but it's far from a solved problem. As the treated population grows and ages, the healthcare system must evolve to meet their lifelong, multifaceted needs.
