Activity Overview
Spinal muscular atrophy (SMA) is a genetic motor neuron disorder that imposes a lifelong physical, emotional, psychosocial, and financial burden on patients and their families. Although disease-modifying therapies have fundamentally changed the treatment landscape, these therapies are not curative, and many treated patients continue to experience residual muscle weakness.
In this archived video from the 2026 Carolina Neuromuscular Disease Summit, expert faculty review the burden of SMA and the limitations of the current standard of care, evaluate emerging clinical trial data for therapies that target muscle directly, and apply updated guideline recommendations to individualized, multidisciplinary management plans that optimize function and quality of life for these patients.
Target Audience
This educational activity is directed toward neuromuscular disease specialists, neurologists, specialty nurse practitioners (NPs), specialty physician assistants (PAs), nurses, and other HCPs involved in the care of patients with neuromuscular disorders.
Learning Objectives
Upon successful completion of this activity, you should be better prepared to:
- Identify the unmet needs for patients with SMA
- Evaluate clinical trial data for disease-modifying therapies for spinal muscular atrophy
- Incorporate guideline recommendations into individualized multidisciplinary management plans for patients with spinal muscular atrophy
Nancy L. Kuntz, MD
Professor of Pediatrics and Neurology
Attending Neurologist
Northwestern Feinberg School of Medicine
Ann & Robert H. Lurie Children’s Hospital of Chicago
Chicago, IL
Disclosures: Advisor, Consultant, Fee for Service Recipient: Med Adv Boards: Argenx, Astellas Pharma, Biogen, Catalyst, Genentech, Regenxbio, Sarepta Express (participation for Gene Rx feedback for physicians), Sarepta Therapeutics, Scholar Rock, Solid Biosciences; Grant/Research Funding: AMO Pharma, Argenx, Astellas Pharma, Biogen, Catalyst, Genentech, Regenxbio, Sarepta Therapeutics, Scholar Rock, Solid Biosciences.
The staff of Physicians’ Education Resource®, LLC, have no relevant financial relationships with ineligible companies.
PER® mitigated all conflicts of interest for faculty, staff, and planners prior to the start of this activity by using a multistep process.
Off-Label Disclosure and Disclaimer
This accredited continuing education activity was planned in accordance with the ACCME Standards for Integrity and Independence to ensure balance, objectivity, independence, and scientific rigor. Faculty were instructed to use generic names when possible and to base recommendations on the best available evidence. Proprietary product names may be referenced in clinical discussion for educational purposes only and do not imply endorsement.
This activity may include discussion of investigational, unapproved, or off‑label uses of drugs or devices. Learners are advised to consult prescribing information for products discussed. Content is provided for educational purposes only and is not a substitute for the independent clinical judgment of a health care professional. Learners are encouraged to critically evaluate the information presented and apply their own clinical judgment.
The views expressed are those of the individual faculty members and do not necessarily reflect the views of the accredited provider or any entity providing commercial support.
This activity may have used AI-assisted tools to support educational development. All content was reviewed and approved by qualified faculty or planners. The accredited provider retains full responsibility for accuracy, balance, and independence.
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