Why Insurance Coverage Has Become a Multidisciplinary Care Problem in Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a rare autosomal recessive neuromuscular disorder caused by biallelic variants in the survival motor neuron 1 (SMN1) gene.¹ Although it is classified as a neurologic condition, its effects extend well beyond the nervous system to respiratory function, nutrition, and bone health. The approval of nusinersen (Spinraza; Biogen) in December 2016, followed by onasemnogene abeparvovec (Zolgensma; Novartis) and risdiplam (Evrysdi; Roche), has added years of life and years of functional life for patients, and newborn screening has moved diagnosis earlier for a growing share of them.^2-4

One consequence is that patients with SMA now reach adulthood nearly universally, which has shifted where the difficulties in their care sit. Rather than the availability of treatment, the pressing question for many families has become whether the specialists who managed a patient through childhood have adult counterparts willing and able to take them on, a question that often turns on insurance coverage rather than clinical need.

W. Bryan Burnette, MD, is chief of neurology at Nemours Children’s Health in Jacksonville, Florida, and a child neurologist and neuromuscular specialist who has cared for patients with SMA for roughly 20 years. He completed his child neurology residency and a neuromuscular medicine fellowship at Johns Hopkins, and his clinical and research interests include the development of novel therapies and biomarkers for inherited neuromuscular disorders.

In recognition of SMA Awareness Month, held annually throughout August, NeurologyLive^® spoke with Burnette about which specialties he considers essential to an SMA care team, why physical therapy and rehabilitation medicine have grown more central as patients age, and how Medicaid coverage gaps in adult care have become one of the field’s most persistent problems. He also reflected on how the treatment landscape has changed across his career.

NeurologyLive: What does an effective multidisciplinary care team look like for a patient with SMA, and which subspecialties do you consider essential?

W. Bryan Burnette, MD: Although SMA is typically classified as a neurologic disease, it really is a multisystem disorder. It not only affects the nervous system, it also affects nutrition, respiratory function. It affects bone health, and so an effective multidisciplinary team should be able to address all of those needs. Often there are also psychosocial needs, as there can be with any chronic medical condition.

So for me, the absolute must-have specialties include neurology, because we’re often guiding the treatment; pulmonary medicine, because even patients who are undergoing treatment can still have respiratory problems; and nutrition. I would also add to that physical therapy, and if available, physical medicine and rehabilitation, to help the patients with adaptive needs.

How have disease-modifying therapies changed the landscape of care, and how has that shifted the roles and priorities of the care team over time?

The disease-modifying therapies have been very successful at adding years of life and years of functional life to individuals living with SMA. As many of those patients are reaching older ages, the role of physical therapy and, as I mentioned earlier, physical medicine and rehabilitation has become increasingly important.

In part that is because 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. That has really become a critical aspect of multidisciplinary care for SMA.

What are some common gaps or challenges you see in coordinating care across subspecialties, and how might clinicians address them?

One of the biggest challenges that I see is that as patients with SMA are living longer and reaching adulthood virtually universally now, continued access to care in adulthood has become a bigger challenge.

Many patients with SMA have Medicaid insurance plans that cover their needs very well through childhood and adolescence, and eventually in adulthood. But many times the adult versions of the specialties that have been providing care don’t accept the Medicaid plans that the patients have. This is a challenge with every specialist that I’m familiar with.

Really, the 3 most important things we can do are advocate, advocate, advocate. Advocate with payers, advocate with adult specialties, to minimize gaps in care. It’s a very challenging role to play, but it’s incredibly important for us to do for every patient every day.

Why is it important to raise awareness of SMA in the neurology community and in the clinic?

As the population of individuals with SMA survives longer, and as more of them receive disease-modifying therapies, the recognition in the community of the need for access to care and access to services has grown tremendously. We have the job of educating our colleagues who perhaps are not as highly specialized as we in our field are, but will be seeing those patients. Whether they’re living in an area where they don’t have a specialty clinic, and they travel a long distance for regular visits that occur relatively infrequently, needing to have someone closer to home who can help with those needs is really important as time goes on, and as the population grows and ages.

Is there anything else you would add?

I mentioned earlier that I’ve been providing care for individuals with SMA for almost 20 years, so I’ve seen all of the changes happen with the treatment, with the shifts in the epidemiology of the disease, and birth rates, and so forth. It’s really one of the most incredible stories of my career, and one of the greatest privileges that I’ve had is to be a part of this process and changing the lives of individuals.

I had so many very difficult conversations with families in the early years of my career, because we had so few options to offer other than supportive care. Now we’re in a completely different world. It is unrecognizable, really, for those of us who started before the era of disease-modifying therapies. And the efforts to promote public awareness of the condition, newborn screening, all of those have been a tremendous success. There’s still a long way to go, but the progress that has been made overall in the last decade is incredible.

Transcript edited for clarity. For more perspectives on the multidisciplinary care approach in SMA, click here to read our previous Q&A in this series.

REFERENCES
1. Weng WC, Lee WT, Chien YH, Tsai LK. Updates of spinal muscular atrophy in advanced therapies. J Formos Med Assoc. 2026;125 Suppl 1:S73-S80. doi:10.1016/j.jfma.2025.10.018
2. U.S. FDA Approves Biogen’s SPINRAZA™ (nusinersen), The First Treatment for Spinal Muscular Atrophy. News release. Biogen. December 23, 2016. Accessed August 14, 2026. https://investors.biogen.com/news-releases/news-release-details/us-fda-approves-biogens-spinrazatm-nusinersen-first-treatment
3. FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA). News release. Genentech. February 12, 2025. Accessed August 14, 2026. https://www.businesswire.com/news/home/20250210728175/en/FDA-Approves-Genentech%E2%80%99s-Evrysdi-Tablet-as-First-and-Only-Tablet-for-Spinal-Muscular-Atrophy-SMA
4. FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. News release. FDA. May 24 2019. Accessed August 14, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease