Neurology

Spinal Muscular Atrophy

Expert Roundtables Podcast: Treatment Algorithm and Best Practices in the Management of Spinal Muscular Atrophy

expert roundtables by John Brandsema, MD; Jenna Klotz, MD, MS; Julie A. Parsons, MD

Overview

<p>The treatment landscape for spinal muscular atrophy (SMA) has rapidly advanced with multiple US Food and Drug Administration (FDA)–approved therapies, bringing new hope to patients with this disorder. Clinicians must navigate a complex and varied patient population, balancing emerging data (including on combination therapies) with individualized care needs. Listen to today’s podcast for updates on these SMA management–related topics.</p>

References

Balaji L, Farrar MA, D’Silva AM, Kariyawasam DS. Decision-making and challenges within the evolving treatment algorithm in spinal muscular atrophy: a clinical perspective. Expert Rev Neurother. 2023;23(7):571-586. doi:10.1080/14737175.2023.2218549

Brown L, Hoffman K, Corbo-Galli C, et al. Serial casting for contractures in SMA: consensus derived guidelines for treatment. Front Neurol. 2025;16:1502495. Published correction appears in Front Neurol. 2025;16:1646078.

Giess D, Erdos J, Wild C. An updated systematic review on spinal muscular atrophy patients treated with nusinersen, onasemnogene abeparvovec (at least 24 months), risdiplam (at least 12 months) or combination therapies. Eur J Paediatr Neurol. 2024;51:84-92. doi:10.1016/j.ejpn.2024.06.004

Glascock J, Sampson J, Haidet-Phillips A, et al. Treatment algorithm for infants diagnosed with spinal muscular atrophy through newborn screening. J Neuromuscul Dis. 2018;5(2):145-158. doi:10.3233/JND-180304

Schroth MK, Deans J, Bharucha Goebel DX, et al. Spinal muscular atrophy update in best practices: recommendations for treatment considerations. Neurol Clin Pract. 2025;15(1):e200374. doi:10.1212/CPJ.0000000000200374

Profile

John Brandsema, MD

Associate Professor of Clinical Neurology
Perelman School of Medicine at the University of Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, PA

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Jenna Klotz, MD, MS

Clinical Associate Professor of Neurology and Neurological Sciences
Director, Pediatric Neuromuscular Neurodiagnostic Lab
Stanford University School of Medicine/Stanford Medicine Children’s Health
Palo Alto, CA

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Julie A. Parsons, MD

Professor of Clinical Pediatrics and Neurology
Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders
Codirector, Neuromuscular Clinic
Children's Hospital Colorado
University of Colorado School of Medicine
Aurora, CO