Achieving Improved Outcomes in Children and Infants with Spinal Muscular Atrophy

Program Dates: November 26, 2019 - November 25, 2020
Credits: 1.5 AMA PRA Category 1 Credits™, 1.5 CNE Contact Hours

Achieving Improved Outcomes in Children and Infants with Spinal Muscular Atrophy

Program Overview

This CME/CE activity is derived from content presented at the American Academy of Pediatrics National Conference and Exhibition in New Orleans, LA. The program will outline best practices for achieving improved outcomes in children and infants with spinal muscular atrophy (SMA). The faculty will first delve into the pathophysiology, genetics and diagnostics of SMA, as well as disease progression and potential biomarkers. Recent clinical trial data of both emerged and emerging agents will be reviewed as it relates to clinical decision making for healthcare professionals. Following, the faculty will discuss therapeutic strategies utilizing the interprofessional team, with the goal of improving outcomes and quality of life for SMA patients. This CME/CE symposium will equip the healthcare team with the tools to better treat and manage their patients with spinal muscular atrophy.

Target Audience

Pediatricians, neurologists, primary care physicians, PAs, NPs, and other healthcare providers involved in the management of patients with SMA

Learning Objectives

  1. Understand the rapid disease progression of SMA, as well as the role neurofilament as a potential biomarker for predicting motor function in patients
  2. Analyze the most recent advances in SMA therapeutics
  3. Utilize the interprofessional healthcare team to develop novel treatment strategies to slow disease progression in infants and children with SMA


Claudia A. Chiriboga-Klein, MD, MPH
Professor of Neurology and Pediatrics
Columbia University Medical Center
New York, New York


John F. Brandsema, MD
Assistant Professor of Clinical Neurology
Children’s Hospital of Philadelphia
Philadelphia, Pennsylvania



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

Stephen L. Nelson, Jr, MD, PhD
Section Head, Pediatric Neurology
Associate Professor of Pediatrics, Neurology, and Psychiatry
Tulane University School of Medicine
New Orleans, Louisiana


Disclosures of Conflict of Interest

It is the policy of AcademicCME that all faculty, instructors, and planners disclose real or apparent conflicts of interest relating to the topics of this educational activity.

The faculty reported the following financial relationships or relationships to products or devices they or their spouse/life partner have with commercial interests related to the content of this CE activity:

Faculty Relationship Identified With:
Claudia A. Chiriboga, MD (Course Chair)


Consultant/Advisor: AveXis; Biogen; Cytokinetics, Inc.; Genentech, Inc.; Ionis Pharmaceuticals, Inc.; Roche
John F. Brandsema, MD


Consultant/Advisor: Alexion Pharmaceuticals; AveXis; Biogen; Cytokinetics; Marathon Pharmaceuticals; PTC Therapeutics; Sarepta Therapeutics
Stephen L. Nelson, Jr., MD, PhD


Speaker’s Bureau: BioMarin Pharmaceutical; Supernus Pharmaceuticals; Sunovion Pharmaceuticals, Inc.

Julie A. Parsons, MD


Consultant/Advisor: AveXis; Biogen; Cytokinetics; Genentech, Inc.; Ionis Pharmaceuticals, Inc.; PTC Therapeutics; Roche; Sarepta Therapeutics; Scholar Rock

Planners, Managers, Reviewers

Timothy Hayes, MD, PhD; Kim Cheramie, MSN, RN-BC; Nicole Brestowski and Chelsey Benedek hereby state that they or their spouse/life partner do not have any financial relationships to products or devices with any commercial interests related to the content of this activity of any amount during the past 12 months.

Accreditation Statement

In support of improving patient care, AcademicCME 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.

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Credit Designation Statements

AcademicCME designates this live material for a maximum of 1.50 AMA PRA Category 1 CreditsTM.

AcademicCME designates this live material for a maximum of 1.50 CNE Contact Hours (Provider number P0491).

Financial Support

This activity has been supported by an independent educational grant from Biogen Inc.

Disclosure of Unlabeled Use

This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. AcademicCME and Biogen Inc. do not recommend the use of any agent outside of the labeled indications.


Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications on dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.

Method of Participation

1. Read the learning objectives, accreditation information and faculty disclosures at the beginning of this activity.
2. Complete the Pre-Activity Questions
3. Read or Review the activity content.
4. Complete the Post-Activity Test Questions and Evaluation.
5. Learners who receive a grade of 70% or better on the Post-Activity Test Questions and complete the Evaluation will receive appropriate credit as indicated (CME, CNE, and/or CPE credit).
6. Learners should claim only the credit commensurate with the extent of their participation in the activity.

CE Inquiries/Special Needs

For all CE inquiries or special needs, please contact [email protected].


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