How Gene Replacement Therapy is Changing the Way We Manage Spinal Muscular Atrophy and Other Neurologic Diseases

Innovations in technology, delivery, and vector systems in gene therapy mean that patients with potentially devastating neuromuscular disorders, such as spinal muscular atrophy (SMA), are now candidates for treatment.  As these therapies emerge, clinicians need to understand how they work and associated broader implications for disease management. This supplement reviews the mechanisms by which viral vector systems correct gene defects and corresponding protein deficits, and discusses findings from recent and ongoing clinical trials. In addition, the authors—all experts in SMA—provide commentary on related topics and respond to frequently asked questions about SMA and gene replacement therapy.


This activity has been designed to meet the educational needs of health care professionals involved in the diagnosis, treatment, or management of patients with spinal muscular atrophy.


Upon completion of this activity, participants will be better able to do the following:

  1. Describe the basic principles of gene replacement therapy
  2. Explain approaches including the use of viral vectors in gene replacement therapy to treat neuromuscular diseases
  3. Review emerging gene replacement therapies currently under investigation for neuromuscular diseases


Samiah Al-Zaidy, MD
Pediatric Neuromuscular Specialist
Formerly with Nationwide Children’s Hospital
Columbus, OH

Diana Castro, MD
Professor of Pediatrics, Neurology & Neurotherapeutics
UT Southwestern Medical Center
Dallas, TX

W. David Arnold, MD
Associate Professor of Neurology
Ohio State University
Columbus, OH

Arthur Burghes, PhD
Professor, Department of Biological Chemistry and Pharmacology
Ohio State University
Columbus, OH

Disclosure of Conflicts of Interest

It is the policy of the Elsevier Office of Continuing Medical Education 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 CME activity:

Name of Faculty or PresenterRelationship Identified With:
Samiah Al-Zaidy, MDConsultant/Advisor: AveXis, Inc.
W. David Arnold


Arthur Burghes, PhD

Consultant/Advisor: AveXis, Inc.

Grant/Research Support: AveXis, Inc.,  and  Exicure

Patents: Intrathecal delivery of recombinant adeno-associated virus 9 (20150252384) licensed to AveXis, a patent Antisense Oligonucleotides for Treatment of Spinal Muscular Atrophy (20160355811) issued, and a patent Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease

Diana Castro, MD

Grant/Research Support: Biogen and Sarepta Therapeutics

Speakers Bureau: Biogen

Non-Faculty: Sandy Breslow; Alison Kemp; Bernard M. Abrams, MD; and The France Foundation staff hereby state that neither they nor their spouse/life partners have any financial relationships to products or devices with any commercials interest related to the content of this activity of any amount during the past 12 months.

Financial Support

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

Provider Information

Provided by the Elsevier Office of Continuing Medical Education in collaboration with The France Foundation.

CME Credit (Physicians)

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

The Elsevier Office of Continuing Medical Education designates this enduring activity for a maximum of 1.0 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

CME Inquiries/Special Needs

For all CME inquiries or special needs, please contact

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. The Elsevier Office of Continuing Medical Education, The France Foundation and AveXis, Inc., do not recommend the use of any agent outside of the labeled indications.

Disclaimer: 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.

Course summary
Available credit: 
  • 1.00 AMA PRA Category 1 Credit(s)™
  • 1.00 Non-physician
Course opens: 
Course expires: 

Available Credit

  • 1.00 AMA PRA Category 1 Credit(s)™
  • 1.00 Non-physician
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