Conference Series: Part 2 of 3

Activity Details

Free CME
0.50 AMA PRA Category 1 Credit(s)
Released: August 21, 2018
Expires: August 20, 2019
30 minutes to complete

Accredited By

Target Audience

Neurologists, pediatric neurologists, neuromuscular specialists, pediatricians, genetic counselors, and other clinicians who see patients with spinal muscular atrophy

Learning Objectives

  • Explore the potential implications of gene therapy for SMA
  • Summarize key clinical trial data on available/emerging treatments for SMA

Activity Description

As new therapies emerge for spinal muscular atrophy (SMA) that include gene replacement and gene modifying therapies, clinicians need to consider the expectations for these treatments. Experts, Dr. Eugenio Mercuri, Dr. Richard Finkel, and Dr. Gyula Acsadi, recently discussed these emerging therapies and their implications on the clinical disease state at an international meeting. This activity is a recording of their roundtable discussion with informative slides broken into 3 parts. The first part discusses diagnosis, genetics, and the role of SMN in SMA. Part 2 explains the results of emerging clinical trial data. The final activity is a practical discussion about the implications of these new therapies for this lethal disease. Discussion topics include the durability of new and emerging treatments and changes to the standards of care for patients with SMA. This education is an engaging update on SMA from the leading experts in the field.

Statement of Educational Need

Many clinicians have questions regarding the genetics of spinal muscular atrophy. In addition, carrier screening is not routinely done, despite ACOG recommendations. Routine newborn testing is currently being considered in many states and internationally. Identification of the neuromotor deficits associated with SMA (and other conditions) is often inaccurate or delayed. As new therapies emerge that include gene replacement and gene modifying therapies, clinicians need to consider the expectations of these treatments. Gene replacement therapy is still a mystery to most clinicians. New therapies prompt a reevaluation of current standards of SMA care.


  • Gene splicing modifiers
  • Gene replacement therapy MOA
  • Clinical trial results


Eugenio Mercuri, MD, PhD (Chair)
Professor of Pediatric Neurology
Catholic University of the Sacred Heart
Rome, Italy

Dr. Eugenio Mercuri is a consultant for AveXis, Biogen, F. Hoffmann-La Roche, and IONIS. He is a principal investigator for Biogen/IONIS and F. Hoffmann-La Roche studies.

Richard S. Finkel, MD
Division Chief of Neurology
Pediatric Neurology
Nemours Children’s Hospital
Orlando, Florida, United States

Dr. Richard Finkel is a consultant for AveXis, Biogen, Catabasis, Ionis, Mitobridge, Novartis, and Roche. He receives clinical trial support from AveXis, Biogen, Catabasis, Cytokinetics, Ionis, Lilly, MDA, NIH, ReveraGen, and Summit. Dr. Finkel owns stock in Roche. His partner holds several patents and receives patent licensing royalties from National Jewish Center and Children’s Hospital of Philadelphia.

Gyula Acsadi, MD, PhD
Division Head of Neurology and Rehabilitation
Professor of Pediatric Neurology
Connecticut Children’s Medical Center
University of Connecticut Medical School
Hartford, Connecticut, United States

Dr. Gyula Acsadi serves as a consultant for AveXis, Biogen, and Serepta. He is on the speakers bureau for Biogen and conducts contract research for Biogen, Italfarmaco, and Serepta.

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 Disclosure

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

Accreditation Statement

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

Designation of Credit

The France Foundation designates this enduring activity for a maximum of 0.50 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

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.

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  • There are no fees for participating in and receiving credit for this activity.
  • Review the activity objectives and CME information.
  • Complete the CME activity
  • Complete the online posttest. A score of at least 75% is required to successfully complete this activity. The participant may take the test until successfully passed.
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  • Credit documentation/reporting:
    • If you are requesting AMA PRA Category 1 Credits™ or a certificate of participation—your CME certificate will be available for download.

Statement of Commercial Support

This activity is supported by an educational grant from AveXis, Inc.

Disclaimer Statement

The France Foundation presents this information for educational purposes only. The content is provided solely by faculty who have been selected because of recognized expertise in their field. Participants have the professional responsibility to ensure that products are prescribed and used appropriately on the basis of their own clinical judgment and accepted standards of care. The France Foundation and the commercial supporter(s) assume no liability for the information herein.

Disclosure of Unlabeled Use

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