Advances in the Management of Spinal Muscular Atrophy: Tailoring Treatment and Care Approaches to Improve Outcomes

A continuing medical education activity provided by NAMCP and AAMCN

This activity is an archive from the live session from the 2022 Fall Managed Care Forum. If you participated in the live session, you are not eligible for continuing education credits from this archive.

This activity is valid from March 1, 2023 to March 1, 2024

Instructions for CME/NCPD: Complete the pre-test, listen to the audio and view the slides, complete the post test, complete the evaluation form and hit submit. You will be asked to enter your name and email address on the pre-test, evaluation and post-test. If you close your internet browser without completing the post test, you will have ONE more opportunity to complete. A score of 70% must be achieved on the post test to receive continuing education credits. If you do not pass the post test after two attempts, you will not be eligible to try again. Once you complete the evaluation form and score 70% or higher on your post test, you will automatically be given your certificate.

To print or save your certificate, you will need to click on the “download” button and either print or save.


Audience: This activity is intended for healthcare professionals practicing in managed care environments.

This activity is supported by educational grants from
Biogen and Genentech, A Member of the Roche Group

Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder characterized by progressive skeletal muscle hypotonia affecting a person’s voluntary movements. This disorder is caused by an abnormal or missing gene known as the survival motor neuron 1 gene (SMN1), which is responsible for the production of a protein essential to motor neurons. Without this protein, lower motor neurons in the spinal cord degenerate and die. The most common symptom is symmetric and progressive muscle weakness. SMA is the second most common autosomal recessive disorder worldwide and the most common cause of infant mortality, with an estimated incidence of 1 in 10,000 live births and estimated prevalence of 1 to 2 per 100,000 persons. Treating and managing spinal muscular atrophy requires a collaborative and multidisciplinary approach. There are two main approaches to treat SMA once a diagnosis for has been made, and these include SMN2 modulators and SMN1 gene therapy. SMN2 modulators alter SMN2 messenger RNA to transcribe exon 7 and produce a full-length SMN protein, and SMN1 gene therapy delivers the SMN1 gene directly to DNA via a viral vector. Currently, these treatments help manage the symptoms and prevent complications. The arrival of disease-modifying therapies has made a major impact on the prognosis for patients with SMA. Although there is no clear cure, there are current and emerging treatment options that can be tailored to patients to improve their outcomes and pave the way for achieving treatment goals.

Upon completion of this activity, participants will be able to:

  • Analyze clinical trial data to guide management decisions for spinal muscular atrophy (SMA), including new and emerging disease modifying therapies for SMA as a treatment option

  • Discuss the best diagnostic tools and testing methods to relieve the symptom burden associated with SMA in adolescents and adults

  • Assess different unmet needs in the management of SMA for a clear understanding on how these gaps will be closed with current and emerging treatment strategies

  • Evaluate key clinical evidence on the role of genetic testing for patients with SMA

  • Describe methods to increase awareness of the benefits of providing well-coordinated multidisciplinary care to patients with SMA


Faculty: Julie A. Parsons, MD
Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders
Professor of Clinical Pediatrics and Neurology
University of Colorado School of Medicine
Children’s Hospital Colorado


(Relevant Financial Relationships with Ineligible Companies in the Last 24 Months):
Julie A. Parsons, MD has served as the principal investigator for Biogen, Novartis, Scholar Rock, Biohaven, and PTC Therapeutics. Dr. Parsons has also served as a consultant for Biogen and Novartis.
Planning Committee: Bill Williams, MD has no relevant financial relationships with an ineligible company in the last 24 months to disclose.

Jeremy Williams has no relevant financial relationships with an ineligible company in the last 24 months to disclose.

Jacqueline Cole, RN, MS, CMCN has no relevant financial relationships with an ineligible company in the last 24 months to disclose.

NAMCP and/or the presenter has copyright or has received permissions for use of materials provided in this activity.

Accreditation & Designation
This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of the National Association of Managed Care Physicians (NAMCP) and American Association of Managed Care Nurses (AAMCN). The National Association of Managed Care Physicians is accredited by the ACCME to provide continuing medical education for physicians.

NAMCP designates this enduring material for a maximum of 1 AMA PRA Category 1 credit(s)TM. Each
physician should claim credit commensurate with the extent of their participation in the activity.

The American Association of Managed Care Nurses is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center's Commission on Accreditation.

Nurses who complete this activity and achieve a passing score will receive 1 hour in nursing continuing professional development.

This activity has been approved by the American Board of Managed Care Nursing for 1.0 contact hours toward CMCN recertification requirements.

This activity is supported by educational grants from
Biogen and Genentech, A Member of the Roche Group

NAMCP and/or this website does not provide medical advice, diagnosis or treatment. NAMCP does not endorse or imply endorsement of the content on any linked website. This website is to be used as an informational resource. With any health related concern, consult with your physician or healthcare professional.

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