Program Description
Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease caused by mutations in the DMD gene that disrupt production of dystrophin, a protein involved in muscle cell formation. DMD results in progressive loss of ambulation, respiratory failure, and cardiomyopathy, and is typically fatal in early adulthood. Corticosteroids remain the standard of care; while treatment slows disease progression, it carries significant long-term toxicities and does not address the underlying genetic mutation. Exon-skipping therapies represent a paradigm shift in DMD treatment by using antisense oligonucleotides to restore a truncated but partially functional dystrophin protein, offering a meaningful therapeutic option to patients with specific mutation profiles. Despite this progress, managed care pharmacists and decision makers face a complex landscape: determining patient eligibility across diverse mutation profiles, evaluating clinical evidence for therapies approved under accelerated pathways, navigating coverage and prior authorization considerations, and addressing the substantial cost burden these treatments carry. Step-therapy protocols, prior authorization criteria, and site-of-care policies must be developed and applied consistently, even as postmarketing confirmatory trials continue to mature. Managed care pharmacists are uniquely positioned to shape evidence-based, equitable, and financially sustainable coverage policies for this class. This supplement is designed to inform managed care pharmacists about the molecular basis of exon-skipping therapy, the clinical evidence supporting approved and emerging agents, and practical guidance for formulary evaluation, utilization management, and care coordination. Participants will review payer and health economics literature to support their roles in evidence-based access decisions, effective collaboration with neuromuscular specialty teams, and optimized outcomes for patients with DMD.
Target audience: Managed care pharmacists
Type of activity: Application
Release date: October 9, 2026
Expiration date: October 9, 2027
Learner level: Foundational, Intermediate
Time to complete activity: 2.0 hours
Fee: Free
Educational Objectives
At the completion of this activity, participants will be able to:
- Associate the genetic basis of Duchenne muscular dystrophy (DMD) with its pathophysiology and need for early treatment initiation
- Differentiate the mechanisms of exon-skipping, gene replacement, and non–dystrophin-based modifiers in DMD to ensure accurate therapeutic selection and clinical expectations
- Apply evidence-based utilization management criteria that align with mutation-specific labeling and account for the burden of DMD to optimize coverage and timely access to therapies
Steven Kheloussi, PharmD, MBA, FAMCP
Principal Consultant
Kheloussi Consulting, LLC
Adjunct Professor of Pharmacy Practice
Wilkes University Nesbitt School of Pharmacy
Wilkes-Barre, PA
Stephen Chrzanowski, MD, PhD
Duchenne Program Director
Assistant Professor
Departments of Neurology and Pediatrics
University of Massachusetts Chan Medical School
University of Massachusetts Memorial Hospital
Worcester, MA
Libbi Green, PharmD
Formulary Operations Manager
Judi Rx
New York, NY
Pharmacy Times Continuing Education™ is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This Application-based activity is approved for 2.0 contact hours (0.20 CEU) under the ACPE universal activity number 0290-0000-26-302-H01-P. The activity is available for CE credit through October 9, 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 Biosciences, Wave Life Sciences
Libbi Green, PharmD, has the following relevant financial relationship with an ineligible company to disclose:
Employee of PBM: Formulary Operations Manager – Benefit Configuration Management
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; Alyssa Bradshaw, PharmD, BCOP; Morgan Gwynn, PharmD, BCOP; Sarah Viruet; and Brianna Winters
Any relevant financial relationships listed for these individuals have been mitigated.
At least one anonymous peer reviewer was used as part of content validation and conflict resolution. The peer reviewer has 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.
For questions about this internet CPE activity, please contact:
[email protected]
Privacy Policy and Terms of Use Information:
www.pharmacytimes.org/terms
