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