Release Date: October 31, 2025
Expiration Date: October 31, 2026
Activity Overview
Spinal muscular atrophy (SMA) is a rare progressive neuromuscular disease that profoundly impacts patients and their families. Over the past decade, remarkable advances in genetic testing, disease-modifying therapies, and multidisciplinary care have transformed the outlook for individuals living with SMA. However, timely diagnosis, optimal treatment selection, and coordinated long-term management remain ongoing challenges.
In this interactive educational forum, participants can review the pathophysiology and disease burden of SMA with a focus on early recognition and diagnosis, explore current and emerging therapies and their implications for clinical practice, and survey multidisciplinary care strategies to address motor, respiratory, nutritional, and psychosocial needs. Participants will gain insight into the rapidly evolving treatment landscape and explore strategies for advancing care and improving outcomes for patients and families affected by this rare disease.
This educational activity is an archive of the live virtual symposium held on September 27, 2025.
Target Audience
This educational activity is directed toward neuromuscular disease specialists, neurologists, specialty nurse practitioners, specialty physician assistants, nurses, and other health care providers involved in the care of patients with neuromuscular diseases.
Learning Objectives
Upon successful completion of this activity, you should be better prepared to:
- Identify the burden of disease of spinal muscular atrophy (SMA)
- Analyze clinical trial data for disease-modifying therapies for SMA
- Apply strategies to optimize the transition of care for patients with SMA
Basil T. Darras, MD
Professor of Neurology
Joseph J. Volpe Chair in Neurology
Harvard Medical School
Chief, Division of Clinical Neurology
Director, Neuromuscular Center and SMA Programs
Boston Children’s Hospital
Boston, MA
Disclosures: Ad Hoc Scientific Advisory Board Member: AveXis/Novartis, Biogen, Merck, Roche/Genentech, Sarepta, Scholar Rock, Vertex; Steering Committee Member: Roche MANATEE studies; Data and Safety Monitoring Board Member: Argenx, Lexeo Therapeutics, Vironexis; Research Support: National Institutes of Health/National Institute of Neurological Disorders and Stroke, Slaney Family Fund for SMA, Spinal Muscular Atrophy Foundation, Working on Walking Fund; Grant Support: AveXis, Biogen, Cure SMA, FibroGen, Ionis, PTC Therapeutics, Roche, Santhera, Sarepta, Scholar Rock, Summitt; Royalties: Elsevier, UpToDate
Faculty, Staff, and Planners’ Disclosures
The staff of Physicians’ Education Resource®, LLC have no relevant financial relationships with ineligible companies.
PER® mitigated all COI for faculty, staff, and planners prior to the start of this activity by using a multistep process.
Off-Label Disclosure and Disclaimer
This activity may or may not discuss investigational, unapproved, or off-label use of drugs. Learners are advised to consult prescribing information for any products discussed. The information provided in this accredited activity is for continuing education purposes only and is not meant to substitute for the independent clinical judgment of a health care professional relative to diagnostic, treatment, or management options for a specific patient’s medical condition. The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER® or any company that provided commercial support for this activity.
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