Making a Difference in Patients With Spinal Muscular Atrophy: Improving Diagnosis and Treatment

Released On
May 11, 2021

Expires On
May 11, 2022

Media Type

Completion Time
30 minutes

Gastroenterology, Genetics, Neurology, Nutrition, Pediatrics, Primary Care, Pulmonology

Pediatrics, Pharmacology, Rehabilitation, Respiratory, Nutrition

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This educational activity is jointly provided by AKH Inc., Advancing Knowledge in Healthcare and Spire Learning.

This activity is supported by an educational grant from Biogen Inc.

Credit Available

CME/CE credit provided by AKH Inc., Advancing Knowledge in Healthcare.

  • Physicians — maximum of 0.5 AMA PRA Category 1 Credit(s)
  • Nurses — 0.5 contact hours (which includes 0.5 hour(s) of pharmacology)

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

Target Audience

The target audience for this educational initiative is neurologists, pediatricians, primary care physicians, and advanced practice providers.

Program Overview

Spinal muscular atrophy (SMA) is an inherited condition that places a tremendous burden on patients, families, caregivers, and healthcare providers, with patients experiencing a wide range of complications and symptoms that are detrimental to day-to-day life. Although SMA management has historically consisted of supportive care, the therapeutic landscape has changed dramatically following the recent approval of transformative medications, particularly disease-modifying therapies (DMTs) that improve motor function and survival. In this educational activity, experts in SMA seek to bring clinicians up-to-date by discussing recent advances in screening for SMA, as well as need-to-know information on newer DMTs, such as relevant clinical efficacy and safety data and recommendations for treatment selection.

Learning Objectives

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

  • RECOGNIZE SMA based on signs, symptoms, and results of genetic tests
  • DESCRIBE safety and efficacy data from clinical trials evaluating disease-modifying therapies for SMA
  • SELECT an appropriate disease-modifying therapy approach for patients with SMA



Basil T. Darras, MD (Faculty Chairperson)

Associate Neurologist-in-Chief
Chief, Division of Clinical Neurology
Joseph J. Volpe Chair in Neurology
Harvard Medical School
Boston Children’s Hospital
Department of Neurology
Boston, MA


Julie A. Parsons, MD (Faculty)

Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders
Professor of Clinical Pediatrics and Neurology
University of Colorado School of Medicine
Children’s Hospital Colorado
Aurora, CO


Accreditation Statement

In support of improving patient care, this activity has been planned and implemented by AKH Inc., Advancing Knowledge in Healthcare and Spire Learning. AKH Inc., Advancing Knowledge in Healthcare is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.


Credit Designation


AKH Inc., Advancing Knowledge in Healthcare designates this enduring material for a maximum of 0.5 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Nurse Practitioners

This activity has been planned and implemented in accordance with the Accreditation Standards of the American Association of Nurse Practitioners (AANP) through the joint providership of AKH Inc., Advancing Knowledge in Healthcare and Spire Learning. AKH Inc., Advancing Knowledge in Healthcare is accredited by the American Association of Nurse Practitioners as an approved provider of nurse practitioner continuing education. Provider number: 030803. This activity is approved for 0.5 contact hour(s) (which includes 0.5 hour(s) of pharmacology).

Disclosures of Conflicts of Interest

Accreditor Disclosure of Conflicts of Interest Statement:
It is the policy of AKH Inc. to ensure independence, balance, objectivity, scientific rigor, and integrity in all of its continuing education activities. The planners and faculty must disclose to the participants any significant relationships with ineligible companies whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients or with the commercial supporter of this accredited continuing education activity. Identified conflicts of interest are mitigated by AKH prior to the planners/faculty assuming their role in this accredited continuing education activity.

Basil T. Darras, MD

  • Advisor: Audentes Therapeutics; AveXis; Biogen Inc; Cytokinetics, Inc; Genentech, Inc; Roche; Sarepta Therapeutics, Inc; Vertex Pharmaceuticals, Inc
  • Contracted Research: AveXis; Biogen Inc; Cytokinetics, Inc; FibroGen, Inc; Ionis Pharmaceuticals; PTC Therapeutics; Roche; Santhera Pharmaceuticals; Sarepta Therapeutics, Inc; Scholar Rock, Inc; Summit Therapeutics
  • Steering Committee Member: Roche
  • Data Safety and Monitoring Board: Amicus Therapeutics

Julie A. Parsons, MD

  • Advisor: Biogen Inc; Genentech, Inc; Novartis; Scholar Rock, Inc
  • Contracted Research: Biogen Inc; Novartis; Scholar Rock, Inc
  • Consultant: Biogen Inc; Novartis

Educational Planning Committee:

Staff, Planners, and Reviewers:

Dorothy Caputo, MA, BSN, RN, CE Director of Accreditations/Lead Nurse Planner, and Bernadette Marie Makar, MSN, APRN, BC, NP-C, have nothing to disclose.

Spire Learning: Chris Washburn, PhD, discloses his spouse/life partner who is employed by Janssen Pharmaceuticals, Inc;

Allison Goldberg, MPA; and Lisa Colombo: No relevant financial relationships with any commercial interests.

All of the relevant financial relationships listed for these individuals have been mitigated.

Instructions for Participation and Credit

There are no fees for participating and receiving CME/CE credit for this enduring activity. To receive CME/CE credit participants must:

  • Log in to participate in the activity.
  • Complete the Pretest.
  • You may move forward through the podcast; however, you may not go back to change answers or review audio content until you finish the presentation.
  • In order to receive a CME/CE certificate, you must complete the activity 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.
Certificates will be emailed to the participant.

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 educational activity may include discussion of uses of agents that are investigational and/or unapproved by the FDA. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.


This course is designed solely to provide the health care professional with information to assist in his/her practice and professional development and is not to be considered a diagnostic tool to replace professional advice or treatment. The course serves as a general guide to the health care professional, and therefore, cannot be considered as giving legal, nursing, medical, or other professional advice in specific cases. AKH Inc. specifically disclaims responsibility for any adverse consequences resulting directly or indirectly from information in the course, for undetected error, or through participant’s misunderstanding of the content.

Contact Information

For questions regarding CE credit, contact AKH Inc., Advancing Knowledge in Healthcare at

Privacy Policy

Spire Learning, LLC is committed to protecting the privacy of personal information from our participants and educational collaborators. Spire maintains physical, electronic, and procedural safeguards that comply with federal regulations to protect against the loss, misuse, or alteration of personal information.

Collection and Use of Your Personal Information
The submission of certain personally identifiable information is necessary to award and track the credits participants may earn after completing an activity. Required items include first name, last name, degree, and email address. These items are necessary. Participants are also required to complete an evaluation of each activity. All evaluative information submitted is collected, retained, and used by Spire Learning, LLC to continuously improve the learning experience.

Spire Learning, LLC will not transfer, sell, or share personal information with outside parties or otherwise disclose personal information unless required to in a legal process. Spire will retain your personal data only for the period necessary to accomplish the stated purposes and to comply with applicable laws.

Copyright Statement

Copyright © 2021 by Spire Learning, LLC and its Licensors. All rights reserved. No part of this publication or activity may be reproduced or transmitted in any form, by any means, without prior written permission of Spire Learning, LLC. Spire Learning, LLC and AKH Inc., Advancing Knowledge in Healthcare will not assume responsibility for damages, loss, or claims of any kind arising from or related to the information contained in this publication or activity, including any claims related to the products, drugs, or services mentioned herein.