Grand Rounds: Advances in Duchenne Muscular Dystrophy

Objectives (Educational Content) :

1. Understand the pathophysiology of Duchenne muscular dystrophy. 2. Understand rationale, efficacy, and barriers to gene-targeted therapies such as exon-skipping and gene replacement therapy. 3. Become aware of upcoming changes to the Ohio newborn screen for Duchenne muscular dystrophy and emerging treatment landscape for newly diagnosed patients.

Target Audience:

General pediatricians, family physicians, nurse practitioners, physician assistants, social workers, psychologists, and nurses.

Identified Gap:

The treatment of Duchenne Muscular Dystrophy (DMD) has been rapidly changing over the past ten years. Novel gene and other therapies show promise to slow the inexorable decline in strength and cardiac function associated with DMD. Newborn screening for DMD will be starting soon. Pediatric providers have a gap in knowledge regarding novel therapies for DMD given the rapid evolution of potential treatments in this area.

Estimated Time to Complete the Educational Activity:

1 hour(s)

Expiration Date for CE/CME Credit:

01-31-2025

Method of Participation in the Learning Process:

The learner will view the presentation, successfully complete a post-test and complete an activity evaluation.

Evaluation Methods:

All learners must successfully complete a post-test, as well as an activity evaluation, to claim CE/CME credit.

Disclosure:

The speaker has returned the disclosure form, indicating that the following relevant financial or other relationship with the following commercial interest: Matthew Ginsberg, MD, Kathryn Mosher, MD, Ian Rossman, MD, PhD, Catherine Ward-Melver, MD The following CME Committee/Planning Committee member(s) has the following relevant financial relationships with ineligible companies to disclose: Merk Maria Victorio, MD All relationships have been mitigated.

Accreditation Statement:

Children’s Hospital Medical Center of Akron is accredited by the Ohio State Medical Association to provide continuing medical education for physicians.

CHMCA designates this enduring material activity for a maximum of 1.0 AMA PRA Category 1 Credit TM.  Physicians should only claim the credit commensurate with the extent of their participation in the activity.

Bibliography:

Dongsheng Duan, Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy, Molecular Therapy, Volume 26, Issue 10, 2018, Pages 2337-2356

https://doi.org/10.1001%2Fjamaneurol.2020.1484

https://www.parentprojectmd.org/duchenne-drug-development-pipeline/Pipeline - Parent Project Muscular Dystrophy (parentprojectmd.org)