Medical management of muscle weakness in Duchenne muscular dystrophy

Sarah R. Rivera, Sumit K. Jhamb, Hoda Z. Abdel-Hamid, Gyula Acsadi, John Brandsema, Emma Ciafaloni, Basil T. Darras, Susan T. Iannaccone, Chamindra G. Konersman, Nancy L. Kuntz, Craig M. McDonald, Julie A. Parsons, Carolina Tesi Rocha, Craig M. Zaidman, Russell J. Butterfield, Anne M. Connolly, Katherine D. Mathews

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish (US)
Article numbere0240687
JournalPloS one
Volume15
Issue number10 October
DOIs
StatePublished - Oct 2020
Externally publishedYes

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)
  • Agricultural and Biological Sciences(all)
  • General

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