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- Title
Medical management of muscle weakness in Duchenne muscular dystrophy.
- Authors
Rivera, Sarah R.; Jhamb, Sumit K.; Abdel-Hamid, Hoda Z.; Acsadi, Gyula; Brandsema, John; Ciafaloni, Emma; Darras, Basil T.; Iannaccone, Susan T.; Konersman, Chamindra G.; Kuntz, Nancy L.; McDonald, Craig M.; Parsons, Julie A.; Tesi Rocha, Carolina; Zaidman, Craig M.; Butterfield, Russell J.; Connolly, Anne M.; Mathews, Katherine D.
- 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.
- Subjects
UNITED States; DUCHENNE muscular dystrophy; MUSCLE weakness; MUSCULAR dystrophy; SYMPTOMS; LIFE expectancy
- Publication
PLoS ONE, 2020, Vol 15, Issue 10, p1
- ISSN
1932-6203
- Publication type
Article
- DOI
10.1371/journal.pone.0240687