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)

ACKNOWLEDGEMENT

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

DATE OF RELEASE/EXPIRATION

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

TARGET AUDIENCE

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.

STATEMENT OF NEED/PROGRAM OVERVIEW

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.

EDUCATIONAL OBJECTIVES

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

JOINT ACCREDITATION STATEMENT

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.

PHYSICIAN CONTINUING EDUCATION

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.

ESTIMATED TIME TO COMPLETE

This activity should take approximately 60 minutes to complete.

INSTRUCTIONS TO RECEIVE CREDIT

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.

HARDWARE/SOFTWARE REQUIREMENTS

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

FEE INFORMATION & REFUND/CANCELLATION POLICY

There is no fee for this educational activity.

FACULTY AND DISCLOSURE OF CONFLICTS OF INTEREST

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.

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 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.

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

CONTACT INFORMATION

Accreditation Support:

For additional information about the accreditation of this activity, please visit https://partnersed.com.

Technical Support:

For any technical issues or issues with your CME Certificate, please contact NueroSeriesLive at 877-394-1306 or at Support@NeuroSeriesLive.com.