AI Article Synopsis
- Duchenne muscular dystrophy (DMD) is a serious childhood condition with limited lifetime expectations, and current care guidelines lack updates on new medications and steroid dosing.
- A systematic review and expert feedback were used to create a list of recommendations for DMD therapies, employing a modified Delphi method to gather panelist insights.
- Preferred treatments include deflazacort or weekend dosing of prednisone, and patients with specific mutations should be considered for eteplirsen alongside corticosteroids, highlighting the need to weigh benefits against potential side effects in treatment choices.
Article Abstract
Introduction: Duchenne muscular dystrophy (DMD) is a childhood onset muscular dystrophy leading to shortened life expectancy. There are gaps in published DMD care guidelines regarding recently approved DMD medications and alternative steroid dosing regimens.
Methods: A list of statements about use of currently available therapies for DMD in the United States was developed based on a systematic literature review and expert panel feedback. Panelists' responses were collected using a modified Delphi approach.
Results: Among corticosteroid regimens, either deflazacort or prednisone weekend dosing was preferred when payer requirements do not dictate choice. Most patients with exon 51 skip-amenable mutations should be offered eteplirsen, before or with a corticosteroid.
Discussion: The options available for medical management of the motor symptoms of DMD are expanding rapidly. The choice of medical therapies should balance expected benefit with side effects.
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Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7571693 | PMC |
| http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0240687 | PLOS |
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