TY - JOUR
T1 - Medical management of muscle weakness in Duchenne muscular dystrophy
AU - Rivera, Sarah R.
AU - Jhamb, Sumit K.
AU - Abdel-Hamid, Hoda Z.
AU - Acsadi, Gyula
AU - Brandsema, John
AU - Ciafaloni, Emma
AU - Darras, Basil T.
AU - Iannaccone, Susan T.
AU - Konersman, Chamindra G.
AU - Kuntz, Nancy L.
AU - McDonald, Craig M.
AU - Parsons, Julie A.
AU - Rocha, Carolina Tesi
AU - Zaidman, Craig M.
AU - Butterfield, Russell J.
AU - Connolly, Anne M.
AU - Mathews, Katherine D.
N1 - Publisher Copyright:
Copyright: © 2020 Rivera et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
PY - 2020/10
Y1 - 2020/10
N2 - 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.
AB - 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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U2 - 10.1371/journal.pone.0240687
DO - 10.1371/journal.pone.0240687
M3 - Article
C2 - 33075081
AN - SCOPUS:85093911283
SN - 1932-6203
VL - 15
JO - PloS one
JF - PloS one
IS - 10 October
M1 - e0240687
ER -