Activity Details

Free CME
0.25 AMA PRA Category 1 Credit(s)
Released: October 1, 2020
Expires: September 30, 2021
15 minutes to complete

Accredited By

Target Audience

This activity will target neurologists, pediatricians, and geneticists, as well as other health care providers who may be involved in the management of patients with SMA.

Learning Objectives

  • Explain how gene therapy works

Activity Description

This education describes various approaches to gene therapy in SMA and reviews clinical trials and data, which continues to accumulate.

Statement of Educational Need

The rapid influx of information surrounding emerging treatments for SMA means that there are critical gaps in clinician knowledge and the ability to apply it in practice, creating a need for education to support clinicians who recognize, diagnose, and/or manage patients with SMA.


Sharon Aharoni, MD
Director of SMA Therapies,
Schneider Institute of Child Neurology Children’s Medical Center
Petach Tikva, Israel

Sharon Aharoni, MD, has participated in contract research for AveXis.

Professor Enrico Bertini
Unit of Neuromuscular and Neurodegenerative Disorders
Laboratory of Molecular Medicine
Bambino Gesu' Children's Research Hospital
Rome, Italy

Professor Enrico Bertini has no financial relationships to disclose.

Professor Aviva Fattal-Valevski
Director, Pediatric Neurology Institute
Dana Children's Hospital, Tel Aviv Medical Center
Tel Aviv, Israel

Professor Aviva Fattal-Valevski has no financial relationships to disclose.

Professor Marcondes Cavalcante França Junior
Assistant Professor of Neurology
The University of Campinas
Sao Paulo, Brazil

Professor Marcondes Cavalcante França Junior has participated in advisory boards for AveXis, Biogen, and Roche.

Professor Wolfgang Müller-Felber
Head of Center for Pediatric Neuromuscular Disorders
and Clinical Neurophysiology
Dr. von Hauner Children’s Hospital
Munich, Germany

Professor Wolfgang Müller-Felber has participated in scientific advisory boards for AveXis, PTC, Sanofi, and Roche.

J. Andoni Urtizberea, MD, MSc
Attending Physician
Institut de Myologie
Paris, France

J. Andoni Urtizberea, MD, MSc, has no financial relationships to disclose.

Conflict of Interest Policy/Disclosure Statement

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.

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 0.25 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.

Method of Participation/How to Receive Credit

  • There are no fees for participating in and receiving credit for this activity.
  • Review the activity objectives and CME/CE information.
  • Complete the CME/CE activity.
  • Complete the online posttest. A score of at least 66% is required to complete this activity. The participant may take the test until successfully passed.
  • Complete the CME/CE evaluation/attestation form. The form provides each participant the opportunity to comment on how 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.
  • Credit documentation/reporting:
    • If you are requesting AMA PRA Category 1 Credits™ or a certificate of participation—your CME/CE certificate will be available for download.

Statement of Commercial Support

This activity is supported by an educational grant from AveXis.

Disclaimer Statement/Disclosure of Unlabeled Use

The France Foundation presents this information for educational purposes only. The content is provided solely by faculty who have been selected because of their 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.

Contact Information for Questions About the Activity

If you have questions about this CME activity, please contact The France Foundation at 860-434-1650 or

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