Events Calendar

Understanding Advances in Gene Therapy

Saturday, June 16, 2018

6:00–8:30 AM
Hilton Anatole
Coronado Room, West Wing, Lobby Level
Dallas, TX


Program Description:
Gene replacement therapy is an emerging treatment for spinal muscular atrophy in phase 3 clinical trials. Our experts will explain the latest developments and how gene replacement therapy works in this CME breakfast symposium. With newborn screening in place in several states, understanding emerging therapies is more important than ever. Dr. Burghes and Dr. Mendell will address questions about safety of emerging therapies and what to consider when discussing new options with patients and families.

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

Jerry R. 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

Target Audience:
This activity is intended for neuromuscular specialists.

Statement of 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 for these treatments. Gene replacement therapy is still a mystery to most clinicians. New therapies prompt a reevaluation of current standards of SMA care.

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

  • Interpret the results of genetic testing for SMA
  • Explore potential implications of gene therapy for SMA
  • Summarize key clinical trial data on emerging treatment for SMA, with a focus on gene therapy

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 1.50 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

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

Hilton Anatole

2201 North Stemmons Freeway
Dallas, Texas, 75207

Location Website


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