SMA Horizons: What Is in the Future for SMA Management?


Claudia A. Chiriboga, MD, MPH
Professor of Neurology and Pediatrics
Columbia University
New York, NY

Julie Parsons, MD
Co-Director of the Neuromuscular Clinic
Professor of Clinical Pediatrics and Neurology
Children’s Hospital Colorado
Aurora, CO

Perry Shieh, MD, PhD
Professor of Neurology
UCLA Medical Center
Los Angeles, CA

Provided by: The France Foundation

Target Audience: This activity is intended for multidisciplinary groups of clinicians including neuromuscular specialists, pediatric neurologists, general neurologists, pediatricians, medical geneticists, genetic counselors, and other health care providers who are involved in the management of spinal muscular atrophy (SMA).

Statement of Need: With three therapies now available for spinal muscular atrophy, clinicians want to know the most recent data and feel confident in diagnosing and treating these patients as early as possible.

Educational Activity Learning Objectives
Upon completion of this course, the participants should be able to:

  • Review pathology, genetics, and epidemiology of SMA
  • Explain the importance of genetic testing for SMA
  • Discuss newborn screening and its importance in early diagnosis of SMA
  • Evaluate new and emerging therapies for SMA

Activity Agenda and Faculty 

  • Meet Andrew, a patient with SMA
  • Introduction to SMA-Dr. Perry Shieh (10 minutes)
  • Panel Discussion: Genetics-led by Dr. Perry Shieh (15 minutes)
  • Q&A
  • Andrew’s Story: Diagnosis
  • Panel Discussion on Newborn Screening-led by Dr. Julie Parsons (15 minutes)
  • Andrew’s Story: Treatment
  • Panel Discussion: Clinical Trials-led by Dr. Claudia Chiriboga (20 minutes)
  • Andrew’s Story: Living With SMA
  • Q&A
  • Post-test and evaluation
  • Chat with faculty

Accreditation Statement: The France Foundation is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Credit Designation
The France Foundation designates this live activity 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.

Nurses: Nurses who are certified by the American Nurses Credentialing Center (ANCC) may utilize activities that are certified by ACCME-accredited providers toward their requirement for certification renewal by the ANCC. A certificate of attendance will be provided by The France Foundation, an ACCME accredited provider.


  1. There are no fees for participating in and receiving credit for this activity.
  2. Review the activity objectives and CME/CE information.
  3. Participate in the CME/CE activity.
  4. Complete the CME/CE evaluation form. This provides each participant with the opportunity to comment on how participating in the activity will affect their professional practice; the quality of the instructional process; the perception of enhanced professional effectiveness; the perception of commercial bias; and his/her views on future educational needs.
  5. Credit documentation/reporting: If you are requesting AMA PRA Category 1 Credit(s)™ or a certificate of participation—your CME/CE certificate will be available for download.

Disclosure Policy: In accordance with the ACCME Standards for Commercial Support, The France Foundation (TFF) requires that individuals in a position to control the content of an educational activity disclose all relevant financial relationships with any commercial interest. TFF resolves all conflicts of interest to ensure independence, objectivity, balance, and scientific rigor in all its educational programs. Furthermore, TFF seeks to verify that all scientific research referred to, reported, or used in a CME/CE activity conforms to the generally accepted standards of experimental design, data collection, and analysis. TFF is committed to providing learners with high-quality CME/CE activities that promote improvements in health care and not those of a commercial interest.

Activity Staff Disclosures: The planners, reviewers, editors, staff, CME committee, and other members at The France Foundation who control content have no relevant financial relationships to disclose.

Faculty Disclosures The following faculty report that they have relevant financial relationships to disclose:

  • Dr. Claudia Chiriboga receives funding support from AveXis, Biogen, NIH, Roche, and SMA Foundation. She is also on the advisory board for AveXis, Cytokinetics, Genentech, PTC Therapeutics, and Roche.
  • Dr. Julie Parsons does contract research for AveXis, Biogen, Scholar Rock, and PTC Therapeutics
  • Dr. Perry Shieh is a consultant for AveXis, Biogen, Genentech, Sarepta, and PTC Therapeutics. He serves on the speakers bureau for Alexion, AveXis, Biogen, CSL Behring, Genentech, and Grifols.