Building a Multidisciplinary SMA Clinic: Lessons From 2 Decades of Practice

When nusinersen (Spinraza; Biogen) was approved in December 2016,¹ it became the first therapy of any kind for spinal muscular atrophy (SMA), a rare autosomal recessive neuromuscular disorder caused by biallelic variants in the survival motor neuron 1 (SMN1) gene. Since then, two additional SMN-enhancing therapies approved followed, including onasemnogene abeparvovec (Zolgensma; Novartis) and risdiplam (Evrysdi; Roche).^2,3

The treatment landscape has continued to expand, with a tablet formulation of risdiplam approved in February 2025,⁴ an intrathecal formulation of onasemnogene abeparvovec (Itvisma) approved in November 2025,⁵ and a high dose regimen of nusinersen approved in March 2026.⁶ Additionally, a decision on apitegromab (Scholar Rock), an investigational myostatin inhibitor for SMA, by the FDA is expected by September 30, 2026.⁷

That expansion has changed what clinicians can offer, but it has not resolved the question of where patients receive care. Coordinated multidisciplinary management has long been identified as central to treatment in SMA, spanning physical and occupational therapy, respiratory care, nutrition, speech-language pathology, and psychosocial support. Assembling and sustaining this type of care team, particularly outside academic centers, remains a practical barrier, and one that determines whether patients reach the clinicians equipped to treat them.

Sandeep Rana, MD, is a neurophysiologist and director of the ALS Center at the Allegheny Health Network Neuroscience Institute, where he also serves as program director of the neurology residency program and as a professor of neurology at Drexel University. His multidisciplinary clinic, established for amyotrophic lateral sclerosis (ALS) and later expanded to serve patients with SMA, has operated for close to 2 decades.

In recognition of SMA Awareness Month, held annually throughout August, NeurologyLive^® spoke with Rana about how his clinic is structured, how the arrival of disease-modifying therapies shifted the team’s priorities toward wellness and long-term function, and why he considers the assembly of a multidisciplinary team an organizational problem as much as a clinical one. He also discussed the specific difficulty of staffing intrathecal administration, and what he would ask of neurologists practicing in community settings.

NeurologyLive: What does an effective multidisciplinary care team look like for a patient with SMA, and what does that process look like when a patient is initially diagnosed?

Sandeep Rana, MD: Care is best rendered for SMA in the form of a multidisciplinary clinic. In this type of clinic, we typically have a physical therapist and an occupational therapist, and they focus on adaptive equipment for the patients if they have developed weakness. We also have respiratory therapists who will track the breathing. Every time a patient comes, their respiratory status is closely monitored, and we assess the need for any interventions like BiPAP machines or CPAP.

Then we also have a nutritionist and a speech therapist monitoring the swallowing process, or if they end up with feeding tubes, what formulation works best for the patient. We also have a social worker addressing all the social issues and providing some counseling as well. In our clinic, we have now also formed a link with one of our pulmonologists who has expertise in neuromuscular disorders. The most effective care can be rendered by this multidisciplinary approach, where a patient comes for one visit and is evaluated by all the necessary disciplines, and we all put our heads together and come up with a unified plan of treatment.

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?

It’s been a very exciting time to be involved in SMA care. Now we have multiple disease-modifying therapies available for SMA. The first one, nusinersen, was FDA approved a decade ago. It has to be administered intrathecally and has proven to be quite effective in modifying the progression of the illness. After that, there has been an oral medication as well, risdiplam, and gene therapy, which is also making a mark now.

With these effective medicines, we have been successful in stabilizing the progression of the weakness in patients, and emphasis has become more on wellness: optimal nutrition, weight control, physical fitness, emotional wellness. But we continue to monitor them closely and make adjustments as needed.

What is still a common gap or challenge you see in coordinating care across the different subspecialties, and how might clinicians help address it?

It is an organizational challenge to bring together this well-trained team who are comfortable managing these types of patients and bring them together in a clinic on a regular basis. I think once you overcome that challenge, it is difficult to replicate. I will say in many of the institutions, it’s hard to bring together such a select group of specialists.

But once that is done, the other issue with providing care for SMA is the need for nursing. One of the very effective drugs has to be administered intrathecally, which is a challenge in itself, to find a specialist who is very well trained and skilled at providing and doing the spinal tap and doing the intrathecal administration of the medication. So it is a challenge, but it’s certainly important for adequate care of our patients.

We’ve been successful in putting together a multidisciplinary clinic. We’ve been running the clinic at Allegheny Health Network for close to 2 decades now. Initially, it was set up for ALS, but with treatments being made available for SMA, this has also become another area where we have provided care.

Why is it important to raise awareness about this condition, and what advice would you offer clinicians?

Not long ago, when I first came out in practice, SMA was considered a fatal illness. There was nothing to offer. But now, with these effective disease-modifying therapies becoming available, I think it’s very important for neurologists out there in the community hospitals to identify cases and refer them in a timely fashion to these tertiary care centers, where we can provide these cutting-edge treatments as well as provide the multidisciplinary care that is optimal for these patients to do well.

So the key is to get the word out, make neurologists aware that there are effective medicines available, and which are the centers that are providing the necessary care. This boils down to services like NeurologyLive, to make everyone aware how fast the landscape is changing, and of ongoing research in SMA. I think everything is looking very optimistic to make further gains.

Is there anything else you would add on SMA?

I think that’s the key thing, that SMA needs to be on the forefront, in the sense that not many neurologists are fully aware of the latest medication treatments that have become available. This is certainly a manageable condition now.

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. U.S. FDA Approves Biogen’s SPINRAZA™ (nusinersen), The First Treatment for Spinal Muscular Atrophy. News release. Biogen. December 23, 2016. Accessed August 10, 2026. https://investors.biogen.com/news-releases/news-release-details/us-fda-approves-biogens-spinrazatm-nusinersen-first-treatment
2. FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA). News release. Genentech. February 12, 2025. Accessed August 10, 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
3. 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 10, 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
4. FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA). News release. Genentech. February 12, 2025. Accessed August 10, 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
5. 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 10, 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
6. FDA Approves New High Dose Regimen of SPINRAZA® (nusinersen) for Spinal Muscular Atrophy. News release. Biogen. March 30, 2026. Accessed August 10, 2026. https://investors.biogen.com/news-releases/news-release-details/fda-approves-new-high-dose-regimen-spinrazar-nusinersen-spinal
7. Scholar Rock Announces FDA Review of Apitegromab Biologics License Application (BLA) for Spinal Muscular Atrophy (SMA) to Progress with Second Fill-Finish Facility; Approval Decision Anticipated by September 30, 2026 Action Date. News release. Scholar Rock. August 7, 2026. Accessed August 10, 2026. https://investors.scholarrock.com/news-releases/news-release-details/scholar-rock-announces-fda-review-apitegromab-biologics-license