Medical management of muscle weakness in Duchenne muscular dystrophy
Year: 2020
Type: 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.
Source: PLoS ONE
Authors: 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
Institutions: University of Pittsburgh, Children's Hospital of Pittsburgh, Connecticut Children's Medical Center, Children's Hospital of Philadelphia, University of Pennsylvania, University of Rochester Medicine, University of Rochester, Boston Children's Hospital, Harvard University, The University of Texas Southwestern Medical Center, University of California San Diego, Lurie Children's Hospital, University of California Davis Medical Center, UC Davis Health, University of Colorado Anschutz, University of Colorado Denver, Stanford University, Washington University in St. Louis, University of Utah, Nationwide Children's Hospital, University of Iowa
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $1,695
Funder: Sarepta Therapeutics
OpenAlex ID: W3093086069