High-Stakes Access: Aligning Managed Care Policy With the Urgency of Treatment in Duchenne Muscular Dystrophy

Program Description

Duchenne muscular dystrophy (DMD) is a rare, X-linked recessive disease that typically presents in early childhood and primarily affects males. DMD is characterized by progressive muscle weakness and degeneration that can lead to decreased quality of life and complications involving the respiratory, orthopedic, and cardiac systems. The pathophysiology of DMD involves frame-shift mutations, most often the deletion of one or more exons in the dystrophin gene, which result in the absence or insufficiency of functional dystrophin protein required to keep muscle cells intact. The therapeutic landscape is rapidly evolving with a focus on disease modification, particularly through exon-skipping therapies that aim to restore the genetic reading frame to produce a functional protein. This session will specifically review underlying genetic mechanisms in DMD and examine the clinical evidence supporting both approved and emerging exon-skipping therapies. After participating, pharmacists and managed care decision-makers will be better equipped to apply best practices for patient-centered care, facilitate timely access to appropriate treatments through informed coverage and formulary decisions, and coordinate multidisciplinary management to mitigate the clinical and economic burden of DMD.

Target audience: Managed care pharmacists
Type of activity: Application
Release date: April 30, 2026
Expiration date: April 30, 2027
Learner level: Foundational, Intermediate
Time to complete activity: 1.5 hour
Fee: Free

Educational Objectives

At the completion of this activity, participants will be able to:

• Explain the underlying genetic and physiological mechanisms of Duchenne muscular dystrophy (DMD), including the role of dystrophin and progressive muscle degeneration
• Analyze the evidence-based treatment recommendations for patients with DMD and the data supporting the use of new and emerging exon-skipping therapies
• Explore best practices to support patient-centered treatment, coordination of care, and access to timely, appropriate therapies for patients with DMD

Accreditation

Pharmacy Times Continuing Education™ is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This activity is approved for 1.5 contact hours (0.15 CEU) under the ACPE universal activity number 0290-0000-26-082-H01-P. The activity is available for CE credit through April 30, 2027.

Instructions for Completing the Activity and Receiving CPE Credit

To receive CPE credit, participants must complete the pretest, view the activity in its entirety, complete the posttest, and complete the online activity evaluation. After successful completion of the online activity evaluation, you can submit your credit to CPE Monitor. You may view your credit within 48 hours at www.mycpemonitor.net. All participants must request credit before the activity expiration date. CE credit will not be issued after this date.

Faculty

Stephen Chrzanowski, MD, PhD, has the following relevant financial relationships with ineligible companies to disclose:

Consultant: Catalyst Pharmaceuticals, ITF Therapeutics, Solid Therapeutics, Wave Life Sciences

The following contributors have no relevant financial relationships with ineligible companies to disclose:

Faculty

Steven Kheloussi, PharmD, MBA, FAMCP

Pharmacy Times Continuing Education™ Planning Staff—Jim Palatine, RPh, MBA; Dipti Desai, PharmD, MBA, CHCP; Liza Patel, PharmD, RPh; Rose Buszka, PharmD, BCPS, CHCP; Adam C Welch, PharmD, MBA, FAPhA; Amy L. Morris, PharmD, BCOP; Daphne Davis, PharmD, BCOP; Katie Naspo; Susan Pordon; Brianna Winters; and Chloe Taccetta have no relevant financial relationships with ineligible companies to disclose.

Educational Disclaimer

Continuing professional education activities are offered solely for educational purposes and do not constitute any form of professional advice or referral. Discussions concerning drugs, dosages, and procedures may reflect the clinical experience of the author(s) or be derived from the professional literature or other sources and may suggest uses that are investigational in nature and not approved by the labeling or indications. Participants are encouraged to refer to primary references or full prescribing information resources.

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