Ontology highlight
ABSTRACT:
SUBMITTER: Sherlock SP
PROVIDER: S-EPMC9294028 | biostudies-literature | 2022 Aug
REPOSITORIES: biostudies-literature
Sherlock Sarah P SP Palmer Jeffrey J Wagner Kathryn R KR Abdel-Hamid Hoda Z HZ Bertini Enrico E Tian Cuixia C Mah Jean K JK Kostera-Pruszczyk Anna A Muntoni Francesco F Guglieri Michela M Brandsema John F JF Mercuri Eugenio E Butterfield Russell J RJ McDonald Craig M CM Charnas Lawrence L Marraffino Shannon S
Journal of neurology 20220408 8
Duchenne muscular dystrophy (DMD) is a progressive, neuromuscular disorder caused by mutations in the DMD gene that results in a lack of functional dystrophin protein. Herein, we report the use of quantitative magnetic resonance imaging (MRI) measures as biomarkers in the context of a multicenter phase 2, randomized, placebo-controlled clinical trial evaluating the myostatin inhibitor domagrozumab in ambulatory boys with DMD (n = 120 aged 6 to < 16 years). MRI scans of the thigh to measure muscl ...[more]