Provider and Caregiver Connection™: Living with Spinal Muscular Atrophy (SMA): Day to Day Patient Care

Released On
July 31, 2019

Expires On
July 31, 2020

Media Type

Completion Time
90 minutes

Neurology, Primary Care, Physiatry


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This activity is provided by Physicians’ Education Resource®, LLC.


Supported by an educational grant from Biogen.

Credit Available

  • Physicians — maximum of 1.5 AMA PRA Category 1 Credit(s)
  • Physicians — maximum of 1.5 contact hours

All other healthcare professionals completing this course will be issued a statement of participation.

Target Audience

This activity is intended for community pediatricians and neurologists, as well as physician assistants, nurse practitioners, primary care physicians, and nurses, involved in the diagnosis, treatment, and management of patients with SMA. This activity will also provide education to a broad multidisciplinary team, who are also invited to participate.

Program Overview

Spinal muscular atrophy (SMA) is a genetic disorder of the spinal motor neuron, with the majority of cases (95%) due to a mutation or deficiency in the SMN1 (survival motor neuron) gene on chromosome 5q13. This type of SMA occurs in 1 out of 11,000 births and is the primary genetic cause of death in infants. Spinal muscular atrophy is classified into 4 phenotypic groups based on age of onset and motor function; the earlier the age of onset, the greater the impact on motor function. Symptoms primarily involve the voluntary muscles, with those most affected closer to the central portion of the body, such as the shoulders, hips, thighs, and upper back. Complications can occur in which breathing and swallowing functionality is affected. Currently, there is no cure for SMA. Clinical management is primarily supportive care led by a pediatrician or neurologist using a vast array of healthcare disciplines, including physical therapists, nurses, rehabilitation specialists, speech and occupational therapists, pulmonologists, orthopedists, and nutritionists. Early diagnosis is imperative. Initiating care early in the course of disease may help to slow progression and loss of function.

This educational activity begins with a mother and caregiver’s story of her 2 daughters with different forms of SMA—from diagnosis and enrollment in clinical trials to an ongoing journey of challenges and successes. Two expert faculty then build on this caregiver perspective to discuss the pathophysiology and genetics involved in SMA, and how these translated into new treatments that can offer improved quality of life and hope for the future. Faculty conclude with strategies for multidisciplinary collaboration to optimize care of patients with SMA.

Benefits of Participating

  • Gain an understanding of SMA through a caregiver’s narrative
  • Recognize the different clinical phenotypes of SMA and the role of molecular genetic testing in diagnosis
  • Learn the impact of antisense oligonucleotides and gene therapy on patient care
  • Review multidisciplinary strategies for individualized disease management

Learning Objectives

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

  • Recognize therapeutic targets in SMA and the mechanisms of action of the various pharmacologic agents
  • Identify the clinical signs of SMA and integrate diagnostic testing along with the pathophysiology to better understand the disease and to initiate patient management
  • Utilize knowledge of the adverse reactions and toxicities of SMA therapeutic options
  • Apply patient perspectives to clinical practice, facilitating informed decision-making


John Brandsema, MD

Neuromuscular Section Head and Neuromuscular Education Director
Children’s Hospital of Philadelphia
Assistant Professor of Clinical Neurology
Perelman School of Medicine at the University of Pennsylvania
Philadelphia, PA

Claudia A. Chiriboga, MD, MPH, FAAN

Professor of Neurology and Pediatrics
Division of Pediatric Neurology, Department of Neurology
Columbia University Medical Center
Director, Pediatric MDA Care Clinic
Pediatric Neuromuscular Division
New York, NY

Gina Cannady (Caregiver Interviewee)

Accreditation Statement

Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians

Credit Designation

Physicians’ Education Resource®, LLC, designates this enduring material for a maximum of 1.5 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Physicians’ Education Resource®, LLC, is approved by the California Board of Registered Nursing, Provider #16669, for 1.5 Contact Hours.

Disclosures of Conflicts of Interest

John Brandsema, MD

  • Grant/Research Support: Biogen, AveXis, PTC Therapeutics, Sarepta, Alexion, Cytokinetics, Pfizer, FibroGen
  • Consultant: Biogen, AveXis, Cytokinetics
  • Speakers Bureau: Biogen

Claudia A. Chiriboga, MD, MPH, FAAN

  • Grant/Research Support: Roche, AveXis, Biogen
  • Consultant: Genentech, Roche, AveXis
  • Speakers Bureau: Biogen Educational Speaker

Gina Cannady (Caregiver Interviewee)

  • Biogen Marketing Campaign for Spinraza®

The staff of Physicians' Education Resource®, LLC, (PER®) have no relevant financial relationships with commercial interests to disclose.

Method of Participation

Instructions for This Activity and Receiving Credit

  1. You will need to log in to participate in the activity.
  2. Each presentation may contain an interactive question(s). You may move forward through the presentation; however, you may not go back to change answers or review audio files/content until you finish the presentation.
  3. At the end of the activity, educational content/audio files will be available for your reference.
  4. In order to receive a CME/CE certificate, you must complete the activity.
  5. Complete the Posttest and pass with a score of 70% or higher, complete the Evaluation, and then click on “Request for Credit.” You may immediately download a CME/CE certificate upon completion of these steps.

Course Viewing Requirements

Supported Browsers:
Internet Explorer 8.0+ for Windows 2003, Vista, XP, Windows 7, Windows 8.1 and above
Google Chrome 28.0+ for Windows, Mac OS, or Linux
Mozilla Firefox 23.0+ for Windows, Mac OS, or Linux
Safari 6.0+ for Mac OSX 10.7 and above

Supported Phones & Tablets:
Android 4.0.3 and above
iPhone/iPad with iOS 6.1 or above.

Disclosure of Unlabeled Use

This CME/CE activity may or may not discuss investigational, unapproved, or off-label use of drugs. Participants are advised to consult prescribing information for any products discussed. The information provided in this CME/CE activity is for continuing medical and nursing education purposes only and is not meant to substitute for the independent clinical judgment of a physician or nurse relative to diagnostic or treatment 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 of the companies that provided commercial support for this program.

Disclosure Policy and Resolution of Conflicts of Interest (COI)

As a sponsor accredited by the ACCME, it is the policy of PER® to ensure fair balance, independence, objectivity, and scientific rigor in all of its CME/CE activities. In compliance with ACCME guidelines, PER® requires everyone who is in a position to control the content of a CME/CE activity to disclose all relevant financial relationships with commercial interests. The ACCME defines “relevant financial relationships” as financial relationships in any amount occurring within the past 12 months that creates a COI.

Additionally, PER® is required by ACCME to resolve all COI. PER® has identified and resolved all COI prior to the start of this activity by using a multistep process.