P33 RKER-065 ameliorated muscle and bone loss in a progressive murine model of Duchenne muscular dystrophy

Patients with Duchenne muscular dystrophy (DMD) have reduced muscle mass and function. They also exhibit low bone strength leading to a higher risk of skeletal fractures. Minimal treatment options include the current standard of care long-term corticosteroids, which further reduce muscle and bone mass. The TGF- β superfamily of ligands, including myostatin and activins, signal through activin type II receptors (ActRII) and negatively regulate muscle and bone mass and function. KER-065 is an investigational, novel modified ActRII-Fc ligand trap designed to target these catabolic ligands.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research