CME Presentations

DMD Gene Therapy



  • John F. Brandsema, MD

    John F. Brandsema, MD

    John F. Brandsema, MD

    Child Neurologist, Neuromuscular Section Head

    John Brandsema, MD, is a child neurologist and the Neuromuscular Section Head at the Children’s Hospital of Philadelphia. He has broad clinical and research interests in neuromuscular diseases, with a particular focus in clinical trials of novel therapies for childhood neuromuscular disorders and is enthusiastic about the promise that medical and scientific advances hold for people with these conditions. Dr. Brandsema is involved with clinical trials of novel therapeutics for neuromuscular disorders. His philosophy of care centers around developing strong partnerships with families and empowering parents to understand their child’s medical condition. Outside of the hospital, he is a classically-trained singer and musician and a budding Philadelphia sports fan.
  • Crystal Proud, MD

    Crystal Proud, MD

    Crystal Proud, MD

    Chief of Neurology and Director of Neuromuscular Medicine

    Crystal Proud, MD is the chief of neurology and director of neuromuscular medicine at Children’s Hospital of the King’s Daughters (CHKD) in Norfolk, Virginia, where she works collaboratively to provide a comprehensive approach to patients with neuromuscular diseases. With her research focused on the development of therapeutic trials for spinal muscular atrophy and Duchenne muscular dystrophy, she is the primary investigator for several clinical trials evaluating novel treatments for patients with various neuromuscular conditions. She also serves as the medical director for the CHKD Novel Therapeutics and Gene Therapy Center, with a commitment to optimizing clinical care and offering participation in clinical research to patients and families affected by rare disease.

CME Information

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Practical Considerations with Gene-Transfer Therapy for Patients with DMD

Jointly provided by Partners for Advancing Clinical Education (PACE) and PlatformQ Health Education, LLC, in collaboration with the National Organization for Rare Disorders (NORD), and American Society of Gene & Cell Therapy (ASGCT)


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


This activity was released on September 15, 2023 and is valid until September 15, 2024. Requests for credit must be made no later than September 15, 2024


The primary target audience for this activity are neurologists, neuromuscular specialists, pediatric neurologists, genetic counselors, and pediatricians. The secondary audience for this activity is primary care physicians.


The approval of a gene-transfer therapy for patients in Duchenne muscular dystrophy (DMD) has brought new hope to some patients with DMD. Understanding the differences between existing and newly available therapies and where and how they fit in the treatment paradigm is crucial to ensuring that they are appropriately leveraged in the care of patients with DMD.

Join a panel of gene-transfer therapy experts to discuss foundational concepts related to gene-transfer therapy for DMD, including data from recent clinical trials explaining the rationale for inclusion/exclusion criteria, and efficacy and safety findings. The panel will also explore the practical considerations around the use of gene transfer therapy, including the importance of patient selection, administration protocols, and multidisciplinary management to optimize care and mitigate or address adverse events.


After completing this activity, the participant should be better able to:
  • Identify the rationale for gene-transfer therapies in the treatment of DMD
  • Describe the latest safety and efficacy data on gene- transfer therapies in DMD, including functional and non-functional outcomes
  • Summarize the multidisciplinary approach to care prior to, during, and after dosing of gene-transfer therapy to ensure appropriate adverse-event management and patient counseling



In support of improving patient care, this activity has been planned and implemented by Partners for Advancing Clinical Education (PACE) and PlatformQ Health Education, LLC. PACE is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.


PACE 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.


This activity should take approximately 60 minutes to complete.


In order to receive credit for this activity, the participant must (1) read the target audience, learning objectives, and disclosure statements, (2) complete the educational activity online, and (3) complete the post-test and activity evaluation. To receive AMA PRA Category 1 Credits™, participants must receive a score of 75% on the post-test.


Participants will need a computer with a recent version of Adobe Flash installed, as well as an internet connection sufficient for streaming media.


There is no fee for this educational activity.


PACE requires instructors, planners, managers, and other individuals who are in a position to control the content of this activity to disclose all financial relationships they may have with ineligible companies. All relevant financial relationships are thoroughly vetted and mitigated according to PACE policy. PACE is committed to providing learners with high-quality accredited CE activities and related materials that promote improvements or quality in healthcare and not a specific proprietary business interest of an ineligible company.

The faculty reported the following relevant financial relationships or relationships to products or devices they have with ineligible companies related to the content of this educational activity:

Faculty Conflicts of Interest
John Brandsema, MD
Neuromuscular Section Head
The Children's Hospital of Philadelphia
Associate Professor of Clinical Neurology
Perelman School of Medicine
University of Pennsylvania
Consultant/Advisory Board: Alexion, Astellas, Biogen, Fibrogen, Genentech/Roche, Janssen, Novartis, Pfizer, PTC Therapeutics, Sarepta, Scholar Rock, Takeda
Researcher: CSL Behring
Crystal M. Proud, MD
Director of Neurology and Neuromuscular Medicine
Children's Hospital of The King's Daughters
Assistant Professor of Pediatrics
Eastern Virginia Medical School
Consultant/Advisory Board: Biogen, Sarepta
Researcher: Biogen, Biohaven, Novartis Gene Therapies, PTC Therapeutics, Sarepta, Scholar Rock

The PACE planners and others have no relevant financial relationship(s) to disclose with ineligible companies. The PlatformQ Health Education, LLC planners and others have no relevant financial relationship(s) to disclose with ineligible companies.


This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications.

The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.


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 and/or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.


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