Serum protein and imaging biomarkers after intermittent steroid treatment in muscular dystrophy.

ABSTRACT: Weekly Steroids in Muscular Dystrophy (WSiMD) was a pilot study to evaluate once weekly prednisone in patients with Limb Girdle and Becker muscular dystrophy (LGMD and BMD, respectively). At study endpoint, there were trends towards increased lean mass, reduced fat mass, reduced creatine kinase and improved motor function. The investigation was motivated by studies in mouse muscular dystrophy models in which once weekly glucocorticoid exposure enhanced muscle strength and reduced fibrosis. WSiMD participants provided blood samples for aptamer serum profiling at baseline and after 6 months of weekly steroids. A subset completed magnetic resonance (MR) evaluation of muscle at study onset and endpoint. At baseline compared to age and sex-matched healthy controls, the aggregate serum protein p

SUBMITTER: Willis AB 

PROVIDER: S-EPMC11579281 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature