Updates in Duchenne’s Muscular Dystrophy: A Focus on Emerging Gene Therapy

Released On
December 31, 2021

Expires On
December 31, 2022

Media Type

Completion Time
60 minutes

Genetics, Pediatrics, Neurology

Pediatrics, Rare diseases, Musculoskeletal diseases, Genetic diseases, Gene Therapy

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

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

Credit Available

  • Physicians — maximum of 1.0 AMA PRA Category 1 Credit(s)
  • Physicians — maximum of 1.0 ABIM and ABP MOC

All other health care professionals completing this course will be issued a statement of participation.

Target Audience

This activity is intended for pediatric neurologists, neuromuscular specialists, developmental neurologists, hospital administrators, and clinical care staff. The secondary target audience is general neurologists, pediatricians, geneticists, genetic counselors, physical therapists, and other clinical staff involved in the multidisciplinary management of patients with DMD.

Program Overview

Newborn screening, early diagnosis, and genetic testing of known carriers for DMD are not happening. It is critically important to recognize and diagnose DMD as early as possible, because earlier treatment has the potential to double the lifespan. Clinical trials for gene therapy in DMD are starting to show encouraging results. The rapid advancement of the field dictates continued education of patients, HCPs, payors, and policymakers on the progress that has been made since early trials. This program will explain the genetic causes of DMD, the natural history of the disease, and how gene therapies may be used to treat DMD. Data from three clinical trial studies are reviewed.

Learning Objectives

Upon completion of this activity, participants should be better able to:

  • Review the genetic underpinnings of DMD
  • Describe the association of gene transfer therapy components: its promoter, transgene and vectors, the outcomes of expression, function, and safety, respectively
  • Compare expression, function, and safety outcomes data for the gene therapies presently under investigation for DMD
  • Evaluate optimal approach to the assessment of gene therapy, related clinical effects, safety, and efficacy




Crystal Proud, MD

Pediatric Neurologist
Children's Hospital of The King's Daughters
Norfolk, Virginia


Perry Shieh, MD, PhD

Professor of Neurology
UCLA Medical Center
Los Angeles, California


Physician Accreditation Statement

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

Physician Credit Designation

The France Foundation designates this enduring activity for a maximum of 1.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Maintenance of Certification
Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 MOC Medical Knowledge point(s) in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC point(s) equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit. Aggregate participant data will be shared with commercial supporters of this activity.

Disclosures of Conflicts of Interest

Crystal Proud, MD
  • Non-CE consulting: Sarepta
  • Contract research: Astellas, Catabasis, Pfizer, PTC, and Sarepta
Perry Shieh, MD, PhD
  • Speaker’s Bureau: Alexion, Avexis, Biogen, CSL Behring, Genentech, and Grifols
  • Non-CE Consulting: Alexion, Avexis, Biogen, Genentech, and Sarepta

Instructions for Participation and Credit

There are no fees for participating and receiving CME credit for this enduring activity. To receive CME credit participants must:

  1. Read the CME/CE information and faculty disclosures.
  2. Participate in the online activity.
  3. Submit the evaluation form.
Certificates will be emailed to the participant

Course Viewing Requirements

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Supported Phones & Tablets:
Android 11.0.0 and above
iPhone/iPad with iOS 12.0 or above

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.


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.

Contact Information

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