Advances in the Spinal Muscular Atrophy Treatment Landscape: Optimal Management to Improve Patient Outcomes

A continuing medical education activity sponsored by NAMCP and AAMCN.

This activity is an archive from the live session from the 2019 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 January 1, 2020 to January 1, 2021

Instructions for CME/CNE: 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.

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

This presentation is supported by educational grants from
AveXis, Biogen, and Genentech

Spinal muscular atrophy (SMA) is a genetic, autosomal recessive disease, affecting the part of the nervous system that controls voluntary muscle movement. This neuromuscular disease is the second most common lethal autosomal recessive disorder in white populations with an overall incidence of 1 in 10,000 live births and a carrier frequency of about 1 in 50. This disorder is caused by an abnormal or missing gene known as the survival motor neuron gene 1 (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, preventing the body’s muscles from receiving signals from the brain. This can lead to progressive muscle weakness and wasting. SMA type (I, II, or III) is determined by the age of onset and the severity of symptoms. The first step in diagnosing a neuromuscular disease includes an in-office physical examination with questions centered around family history and simple blood tests to indicate whether deletions or mutations of the SMN1 gene are present. Treating and managing SMA requires a collaborative and multidisciplinary approach. Although there is no cure, there are current and emerging treatments that help manage the symptoms and prevent complications. Physical therapy, occupational therapy, and rehabilitation may help to improve posture, prevent joint immobility, and slow muscle weakness and atrophy. One of the more promising treatments involves gene therapy designed to deliver a copy of the SMN1 gene to motor neurons in SMA patients. Once the SMN1 gene reaches patients’ cells, it supplements those cells’ own production of SMN protein.

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

  • Analyze the safety and efficacy data of recently available disease-modifying treatments for spinal muscular atrophy (SMA)

  • Discuss the role of genetics in the diagnosis and management of SMA, including its recessive tendencies and recommendations for genetic screening

  • Explore the evolving role of gene therapy in the management of SMA

  • Assess the role of real-world data to better understand the impact of current and emerging treatments for patients with SMA

  • Evaluate key clinical evidence on newer treatment options for patients with SMA

Faculty: John Brandsema, MD
Attending Physician
Neuromuscular/General Neurology & Electromyography
Neuromuscular Education Director and Neuromuscular Section Head
Children's Hospital of Philadelphia


Dr. Brandsema has served on an advisory board for AveXis and Biogen. He has received grant/research support from Biogen, AveXis, Cytokinetics, PTC Therapeutics, Sarepta, Alexion, Summit, Pfizer, Fibrogen, and WAVE. He serves on the speaker's bureau for Biogen. His presentation has been peer reviewed for any bias.
  Planning Committee:
Bill Williams, MD has no relevant financial relationships to disclose.
Jeremy Williams has no relevant financial relationships to disclose.
Will Williams has no relevant financial relationships to disclose.
Jacqueline Cole, RN, MS, CMCN has no relevant financial relationships to disclose.

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

Accreditation & Designation
The National Association of Managed Care Physicians (NAMCP) is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

NAMCP designates this enduring material for a maximum of 1 AMA PRA Category 1 creditsTM.

The American Association of Managed Care Nurses (AAMCN) 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 continuing
nursing credit.

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

This presentation is supported by educational grants from
AveXis, Biogen, and Genentech

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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