Coordinating Multidisciplinary SMA Care in an Expanding Treatment Landscape

Spinal muscular atrophy (SMA) is a rare autosomal recessive neuromuscular disorder caused by biallelic variants in the survival motor neuron 1 (SMN1) gene, leading to degeneration of motor neurons in the spinal cord and progressive proximal muscle weakness. The approval of 3 SMN-enhancing therapies, including nusinersen (Spinraza; Biogen), onasemnogene abeparvovec (Zolgensma; Novartis), and risdiplam (Evrysdi; Roche), has substantially altered the natural history of the disease, shifting the clinical conversation from survival toward long-term function and quality of life.

That shift has widened rather than narrowed the range of clinicians involved, and the past 18 months have added further complexity. In February 2025, the FDA approved a tablet formulation of risdiplam;¹ in November 2025, it approved an intrathecal formulation of onasemnogene abeparvovec (Itvisma) for patients aged 2 years and older;² and in March 2026, it approved a high dose regimen of nusinersen.³ Consensus standards of care have long identified coordinated multidisciplinary management as central to treatment, spanning neurology, pulmonology, physical and occupational therapy, nutrition, orthopedics, genetic counseling, and psychosocial support. As the number of therapeutic options grows and treated patients live longer, care teams are increasingly navigating questions those guidelines were not written to answer.

Divya Jayaraman, MD, PhD, assistant professor of neurology and pediatrics in the Division of Child Neurology at Columbia University Irving Medical Center, is among the clinicians working through them. At Columbia, she serves as co-director of the pediatric neuromuscular clinic, director of the newborn screening subspecialty care center for pediatric neuromuscular diseases including SMA, associate program director of the pediatric neurology residency program, and a member of the Muscular Dystrophy Association Care Center team. She completed her pediatrics residency, child neurology residency, and neuromuscular fellowship training at Boston Children's Hospital, where she also worked as an SMA clinical trial sub-investigator.

In recognition of SMA Awareness Month, held annually throughout August, NeurologyLive^® spoke with Jayaraman about how her clinic structures its care team and which specialties she considers essential. They also covered how the expansion of disease-modifying therapy (DMT) options has reshaped clinic visits: Jayarama described the challenge of counseling families through an ever-growing menu of treatments with limited comparative data, and how she frames conversations with families who expect a new therapy to reverse existing motor neuron loss. Finally, she discussed why the rest of the care team matters more than ever, as DMT discussions now take up much of the neurologist's time during each visit.

NeurologyLive: What does an effective multidisciplinary care team look like for a patient with SMA, and which specialties do you consider non-negotiable?

Divya Jayaraman, MD, PhD: I think our clinic here, as well as the clinic I trained in at Boston Children’s, is a good model for this. At Boston Children's, I trained with the team, including Basil T. Darras, MD, and everyone over there. The core of our clinic here, I would say, is a hub and spokes model. The pediatric neuromuscular neurologist is a quarterback, but we would not be able to do this without our multidisciplinary team.

We have physical therapists who are key, not just as clinical evaluators, but also for the ongoing monitoring of treatment response with us. In the early diagnostic phases, especially coming out of newborn screening, or symptomatic, if they did not have newborn screening for some reason, the genetic counselor is hugely important, and we have one embedded in our clinic. Once we're past the initial diagnosis phase, their role diminishes, and then other subspecialists become more important. The pulmonologist is especially key, and is also embedded in our clinic. Those would be the essentials. We do have access to orthotists and assistance with equipment, so that's usually coordinated by our physical therapists, and then we have a social worker who's historically been part of our clinic.

Other centers might have more subspecialists. For us in New York, space is at a premium, and there are only so many clinic rooms we can occupy on our SMA and Duchenne muscular dystrophy days. So this is the minimum set that we've arrived at, and then we refer for other needs.

As DMTs have changed the trajectory of SMA, how has that shifted the roles or priorities of the care team over time?

I was reflecting on the first question and realizing that my perspective is very influenced by having trained entirely in the DMT era. I was a third- or fourth-year medical student when nusinersen was approved. All of my training and practice have unfolded in the treatment era.

For our patients who preceded the treatment era, they have way more needs. A lot of them have gastrostomy tubes, they might be wheelchair bound, they might have scoliosis and other orthopedic needs. So those specialists are all very much involved in the care of our patients. They may not be physically embedded in our clinic, and that may be a difference between our clinic and some other multidisciplinary clinics.

The nature of the care also evolves over time. For patients who were identified on newborn screening and presymptomatically treated, that ongoing care looks very different from someone who was maybe a type 1 who was diagnosed over a decade ago and wasn't in any of the trials. We're spanning a huge range there, and we have all of those. We have our type 1 s who've been seeing us for years and years, and then the type 2s and 3s, and then we have the more recently diagnosed and treated, hopefully presymptomatically.

With more therapies available and relatively limited comparative data, what does that add to the clinic visit?

For one, we not only have DMTs, we have options and an ever-expanding number of options, and we have relatively limited comparative data, which makes our role more challenging in many ways. For example, a lot of patients who may have been stably on either risdiplam or on intrathecal nusinersen for a long time are coming back to us and asking, “should we get high dose nusinersen, or should we consider the intrathecal onasemnogene gene therapy?” That's a nuanced discussion, and the answer may be different from patient to patient. It involves going through what do we know from the trial data, what do we not know, what claims can and cannot be made, and then understanding where that all fits into the insurance landscape as well. And that's not even getting into what the risk tolerance is of a given family, not to mention our own risk tolerance.

Just navigating this ever-expanding number of DMTs is our main preoccupation in the clinic right now, I would say. Then the other piece is just keeping up with continuing the existing treatments and all of the prior authorizations, making sure that nothing falls through the cracks.

How do you frame those conversations with families who may expect a newly approved therapy to reverse what has already happened?

We have to recognize that none of these treatments are cures. They may help preserve, to whatever degree possible, the motor neurons that the patient has, and delay and slow disease progression. But for someone who has already lost motor neurons and is symptomatic, type 3, type 2, type 1, we're having to rediscuss that these DMTs are not going to reverse what has already happened. Sometimes that can be quite devastating for families. If they had been on a stable regimen for a while, they see the news, they see the new treatments coming out, and are saying, “oh, is this going to make my child walk again?” And then explaining that that's not necessarily the case.

To be clear, this is still night and day compared with a decade ago. We're in 2026, so over a decade ago, we didn't have anything to offer. So recognizing that both things can be true at once: we have treatments that are time sensitive, and at the same time, once motor neurons are lost, there's not much we can do to turn back the clock on that. The priority becomes, how do we preserve what we have, and how do we optimize function?

I would say the role of the other members of the care team is only more important now, because those considerations are often crowded out in the discussion with the neurologist. Just examining them, getting the updates, and then going through what the new treatment options are takes up most of the visit. So then we rely on the therapist, we rely on the pulmonologist, we rely on the other members of the team to elicit what the other needs are.

Transcript edited for clarity. For more on the multidisciplinary care approach in SMA, read our review of the latest literature.

REFERENCES
1. FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA). News release. Genentech. February 12, 2025. Accessed August 7, 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
2. Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA). News release. Novartis. November 24, 2025. Accessed August 7, 2026. https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-itvisma-only-gene-replacement-therapy-children-two-years-and-older-teens-and-adults-spinal-muscular-atrophy-sma
3. FDA Approves New High Dose Regimen of SPINRAZA® (nusinersen) for Spinal Muscular Atrophy. News release. Biogen. March 30, 2026. Accessed August 7, 2026. https://investors.biogen.com/news-releases/news-release-details/fda-approves-new-high-dose-regimen-spinrazar-nusinersen-spinal