Spinal muscular atrophy (SMA) has moved from a fatal childhood disease to a chronic condition that many patients now live with into adulthood, thanks to the approval of disease‑modifying drugs such as nusinersen, onasemnogene abeparvovec and risdiplam.
What a multidisciplinary team looks like
According to Dr. W. Bryan Burnette, chief of neurology at Nemours Children’s Health, an effective SMA care team must go beyond neurology. “It really is a multisystem disorder,” he said, noting that the condition also impacts nutrition, respiratory function and bone health. The core specialties he highlighted include neurology, pulmonary medicine, nutrition, physical therapy and, when available, physical medicine and rehabilitation. These providers address both medical and psychosocial needs that accompany a lifelong disorder.
Physical therapy and rehabilitation have become increasingly central as patients age. “They’re often the providers in our group who help us the most with guiding our treatment, assessing response to treatment, and allowing patients to continue to receive insurance coverage for their treatment,” Burnette explained. This shift reflects the added functional years that newer therapies provide.
Insurance gaps loom for adult patients
One of the most persistent challenges is the transition from pediatric to adult care. Many individuals with SMA are covered by Medicaid plans that support childhood and adolescent services, but adult specialists frequently do not accept those plans.
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Medicaid’s limitations affect every specialty involved in SMA care, from pulmonology to nutrition. The result can be interruptions in treatment that jeopardize the gains achieved through early intervention. Families often find themselves searching through a fragmented system, looking for providers who will accept their insurance while maintaining the same level of expertise they received as children.
While the medical community has made strides in extending life expectancy, the infrastructure for long‑term support has not kept pace. The need for coordinated adult services is now a priority for clinicians who have watched patients grow from infants to adults over two decades.
Gene‑based and antisense therapies will likely increase the number of adults living with SMA. This demographic shift may pressure health systems to develop dedicated adult SMA clinics, similar to those that exist for other chronic neuromuscular disorders. However, without policy changes or broader insurance acceptance, the risk of care gaps remains high.
Clinicians are encouraged to maintain close communication with families, ensuring that transition plans are established well before patients reach adulthood. By doing so, they can mitigate the disruption that often accompanies the shift to adult providers.