Ontology highlight
ABSTRACT:
SUBMITTER: Rivera SR
PROVIDER: S-EPMC7571693 | biostudies-literature | 2020
REPOSITORIES: biostudies-literature
Rivera Sarah R SR Jhamb Sumit K SK Abdel-Hamid Hoda Z HZ Acsadi Gyula G Brandsema John J Ciafaloni Emma E Darras Basil T BT Iannaccone Susan T ST Konersman Chamindra G CG Kuntz Nancy L NL McDonald Craig M CM Parsons Julie A JA Tesi Rocha Carolina C Zaidman Craig M CM Butterfield Russell J RJ Connolly Anne M AM Mathews Katherine D KD
PloS one 20201019 10
<h4>Introduction</h4>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.<h4>Methods</h4>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 m ...[more]