ARTICLE | Clinical News
N-Gene preclinical data
April 30, 2012 7:00 AM UTC
In mouse models of DMD, once-daily 15 mg/kg oral BGP-15 significantly reduced whole body muscle breakdown as measured by creatine kinase levels vs. untreated controls (p<0.01). Additionally, BGP-15 for 12 weeks significantly reduced damaged myofibers, as measured by Evans blue dye infiltration in myoplasm, vs. untreated controls (p<0.05). In mice with a phenocopy of DMD that results in severe spinal curvature (kyphosis), muscle weakness and premature death, BGP-15 significantly reduced kyphosis (p<0.05), improved the dystrophic pathophysiology in limb and diaphragm muscles (p<0.01 for both) and extended median lifespan by 27% (p<0.05) vs. untreated controls. Data were published in Nature. ...