Activity Details

Free CME
1.50 AMA PRA Category 1 Credit(s)
Released: November 15, 2018
Expires: November 14, 2019
90 minutes to complete

Accredited By

Target Audience

Pediatric Neurologists, Neurologists, Neuromuscular Specialists, Pediatricians

Learning Objectives

  • Understand importance of early diagnosis for inherited neuromuscular diseases
  • Explore potential implications of gene therapy
  • Summarize key clinical trial data on emerging gene therapies
  • Provide appropriate counseling to affected individuals and families

Activity Description

Gene replacement therapy is emerging as a potential treatment for rare CNS diseases. In this recorded CME satellite symposium to the 2018 Child Neurology Society’s annual meeting, a parent of a child with spinal muscular atrophy describes their journey through gene therapy clinical trials. Using spinal muscular atrophy as an example, Dr. Samiah Al-Zaidy, Mr. Jaysson Eicholtz and Dr. Jerry Mendell walk through the history of gene therapy from the bench to the bedside, answering questions on safety and long-term durability of this emerging treatment.

Statement of Educational Need

Gene replacement therapy is showing promising results for several neurologic diseases. Clinicians must be comfortable with this technology before it comes to the clinic. Delayed diagnosis is a common problem with spinal muscular atrophy, even when specialists are involved. Identification of the neuromotor deficits associated with SMA (and other inherited neuromuscular diseases) is often inaccurate or delayed. Prompt diagnosis and treatment is crucial in the effectiveness of gene replacement therapy. These emerging new therapies prompt a reevaluation of current standards of care for patients with SMA and appropriate counseling to the affected individuals and families.


Introduction (Dr. Samiah Al-Zaidy)
Patient Journey Video: Meet the Villarreal family
History of Gene Therapy and the Current Landscape (Dr. Samiah Al-Zaidy)
Patient Journey Video: Diagnosis
Bringing the Vector to the Clinic (Mr. Jaysson Eicholtz)
Patient Journey Video: Treatment
Gene Therapy Clinical Trials: the Epitome of Personalized Medicine (Dr. Jerry Mendell)
Patient Journey Video: Results
Gene Therapy Clinical Trials: Part 2 (Dr. Jerry Mendell)
Panel Discussion (Dr. Samiah Al-Zaidy, Dr. Jerry Mendell, Mr. Jaysson Eicholtz)
Patient Journey Video: Living with SMA
Focus on Patient-Centered Care (Dr. Samiah Al-Zaidy, Dr. Jerry Mendell)


Samiah Al-Zaidy, MD
Pediatric Neuromuscular Specialist
Formerly of Nationwide Children’s Hospital
Columbus, OH
  • Dr. Al-Zaidy serves as a consultant for AveXis, Inc.

Jaysson Eicholtz, MS
Former Director of GMP Operations
Nationwide Children’s Hospital
Columbus, OH
  • Mr. Eicholtz has no relevant financial relationships to disclose.

Jerry Mendell, MD
Curran-Peters Chair of Pediatric Research
Professor of Pediatrics and Neurology
Nationwide Children’s Hospital and
The Ohio State University
Research Institute at Nationwide Children's Hospital
Columbus, OH
  • Dr. Mendell serves as a consultant to AveXis, Inc., Exonics, Myonexus, and Sarepta Therapeutics. He is the principal investigator of the AVXS-101 gene therapy clinical trial, the Eteplirsen clinical trial, and an advisor on the CRISPR clinical trial.

Conflict of Interest Policy/Disclosure Statement

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, 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 1.50 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.

Instructions for Receiving 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 75% is required to successfully complete this activity. The participant may take the test until successfully passed.
  • Complete the CME/CE evaluation/attestation form, which 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.
  • 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, Inc.


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

TFF requires CME faculty (speakers) to disclose when products or procedures being discussed are off label, unlabeled, experimental, and/or investigational, and any limitations on the information that is presented, such as data that are preliminary, or that represent ongoing research, interim analyses, and/or unsupported opinion. Faculty in this activity may discuss information about pharmaceutical agents that is outside of US Food and Drug Administration approved labeling. This information is intended solely for continuing medical education and is not intended to promote off-label use of these medications. TFF does not recommend the use of any agent outside of the labeled indications. If you have questions, contact the Medical Affairs Department of the manufacturer for the most recent prescribing information.

Contact Information

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