Journal article
Medical management of muscle weakness in Duchenne muscular dystrophy
PloS one, Vol.15(10), pp.e0240687-e0240687
2020
DOI: 10.1371/journal.pone.0240687
PMID: 33075081
Abstract
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.
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.
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.
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.
Details
- Title: Subtitle
- Medical management of muscle weakness in Duchenne muscular dystrophy
- Creators
- Sarah R Rivera - Department of Clinical Services, Optum Lifesciences Wolfeboro, Wolfeboro, New Hampshire, United States of AmericaSumit K Jhamb - Department of Clinical Services, Optum Global Solutions, Noida, Uttar Pradesh, IndiaHoda Z Abdel-Hamid - Division of Child Neurology, Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States of AmericaGyula Acsadi - Department of Neurology, Connecticut Children's Medical Center, Farmington, Connecticut, United States of AmericaJohn Brandsema - Division of Neurology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, United States of AmericaEmma Ciafaloni - Department of Pediatric Neuromuscular Medicine, University of Rochester Medical Center, Rochester, New York, United States of AmericaBasil T Darras - Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts, United States of AmericaSusan T Iannaccone - Department of Pediatrics, UT Southwestern, Dallas, Texas, United States of AmericaChamindra G Konersman - Department of Neurosciences, University of California, San Diego, San Diego, California, United States of AmericaNancy L Kuntz - Department of Pediatrics, Ann & Robert H Lurie Children's Hospital, Chicago, Illinois, United States of AmericaCraig M McDonald - Department of Physical Medicine & Rehabilitation, UC Davis Health, Sacramento, California, United States of AmericaJulie A Parsons - Department of Pediatrics and Neurology, University of Colorado School of Medicine, Aurora, Colorado, United States of AmericaCarolina Tesi Rocha - Department of Neurology, Stanford University, Palo Alto, California, United States of AmericaCraig M Zaidman - Department of Neurology, Divisions of Child Neurology and Neuromuscular, Washington, University in St. Louis School of Medicine, St. Louis, Missouri, United States of AmericaRussell J Butterfield - Department of Neurology and Pediatrics, University of Utah, Salt Lake City, Utah, United States of AmericaAnne M Connolly - Department of Neurology, Nationwide Children's Hospital, Columbus, Ohio, United States of AmericaKatherine D Mathews - Departments of Pediatrics, University of Iowa Carver College of Medicine, Iowa City, Iowa, United States of America
- Resource Type
- Journal article
- Publication Details
- PloS one, Vol.15(10), pp.e0240687-e0240687
- DOI
- 10.1371/journal.pone.0240687
- PMID
- 33075081
- NLM abbreviation
- PLoS One
- ISSN
- 1932-6203
- eISSN
- 1932-6203
- Publisher
- Public Library of Science; United States
- Language
- English
- Date published
- 2020
- Academic Unit
- Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
- Record Identifier
- 9984070121902771
Metrics
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