Diagnosis and Treatment of Spinal Muscular Atrophy in an Evolving Therapeutic Landscape
John Brandsema, MD
Assistant Professor of Clinical Neurology
Perelman School of Medicine
University of Pennsylvania
Neuromuscular Section Head
Children's Hospital of Philadelphia
Basil T. Darras, MD
Professor of Neurology
Harvard Medical School
Chief, Division of Clinical Neurology
Director, Neuromuscular Center and Spinal Muscular Atrophy Program
Boston Children’s Hospital
Richard S. Finkel, MD
Professor of Neurology
University of Central Florida College of Medicine
Chief, Division of Pediatric Neurology
Director, Neuromuscular Program
Nemours Children’s Hospital
Julie Blum, PhD
Director, Clinical Content
Laura Rafferty, ELS
Kathryn Schaefer, MSN, RN, CPHRM
Senior Manager, Accreditation and Compliance
East Lansing, MI
Upon completion, participants should be able to:
- Describe symptoms of SMA in an infant or child
- Discuss the benefits of early diagnosis and treatment of SMA
- Understand the role of pediatricians in maintaining best practices for treating SMA
- Describe current newborn screening standards for SMA
This activity is intended for physicians (including pediatricians, pediatric neurologists, and neuromuscular specialists), nurse practitioners, physician assistants, nurses, and other clinicians who care for people with SMA.
Statement of Need
SMA is a rare, autosomal recessive genetic disease characterized by muscle weakness and physical disability attributed to motor neuron degeneration in the spinal cord and brainstem. Early diagnosis is key because it may lead to early treatment initiation; however, delays in diagnosis are common in SMA. In light of the recent United States Food and Drug Administration approvals of therapies for SMA, clinicians must be able to promptly and accurately diagnose this condition. Moreover, there is an active research pipeline that includes several novel disease-modifying therapies that are producing promising results in clinical trials. These agents have the potential to transition to mainstream clinical practice relatively quickly; therefore, clinicians must have a thorough understanding of the efficacy and safety data and potential role of these agents so that they are prepared to incorporate them into treatment regimens as they become available.
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Med-IQ designates this enduring material for a maximum of 1.5 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Nurse practitioners, physician assistants, and other healthcare professionals who successfully complete the activity will receive a Statement of Participation indicating the maximum credits available.
Medium/Method of Participation
This CME activity consists of a 1.5-credit online publication. To receive credit, read the introductory CME material, read the publication, and complete the post-survey, evaluation, attestation, and post-test, answering at least 70% of the post-test questions correctly.
Initial Release Date: January 23, 2020
Expiration Date: January 22, 2021
Estimated Time to Complete This Activity: 1 hour and 30 minutes
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The content of this activity has been peer reviewed and has been approved for compliance. The faculty and contributors have indicated the following financial relationships, which have been resolved through an established COI resolution process, and have stated that these reported relationships will not have any impact on their ability to give an unbiased presentation.
John Brandsema, MD
Consulting fees/advisory boards: AveXis, Biogen Idec, Cytokinectics, Inc., PTC Therapeutics
Fees received for promotional/non-CME activities: Biogen Idec
Contracted research: Alexion Pharmaceuticals, Inc., AveXis, Biogen Idec, Cytokinectics, Inc., FibroGen, Inc., Pfizer, Inc., PTC Therapeutics, Sarepta Therapeutics, Wave
Basil T. Darras, MD
Consulting fees/advisory boards: AveXis, Biogen Idec, Cytokinetics, Inc., Dynacure, F. Hoffmann-La Roche Ltd., Genentech, Sarepta Therapeutics
Richard S. Finkel, MD
Consulting fees/advisory boards: AveXis, Biogen Idec, Cytokinetics, Inc., F. Hoffmann-La Roche Ltd., Genentech, Neurogene Inc., Novartis Pharmaceuticals Corporation, Scholar Rock
Contracted research: AveXis, Biogen Idec, Catabasis Pharmaceuticals, Inc., Cytokinetics, Inc., F. Hoffmann-La Roche Ltd., Italfarmaco S.p.A., ReveraGen BioPharma, Inc., Scholar Rock, Summit Pharmaceuticals International Corporation
The peer reviewers and activity planners have no financial relationships to disclose.
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This activity is supported by an educational grant from Genentech.
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