Francesco Muntoni, Barry J Byrne, Hugh J McMillan, Monique M Ryan, Brenda L Wong, Juergen Dukart, Amita Bansal, Valerie Cosson, Roxana Dreghici, Maitea Guridi, Michael Rabbia, Hannah Staunton, Giridhar S Tirucherai, Karl Yen, Xiling Yuan, Kathryn R Wagner
INTRODUCTION: Duchenne muscular dystrophy (DMD) is a genetic muscle disorder that manifests during early childhood and is ultimately fatal. Recently approved treatments targeting the genetic cause of DMD are limited to specific subpopulations of patients, highlighting the need for therapies with wider applications. Pharmacologic inhibition of myostatin, an endogenous inhibitor of muscle growth produced almost exclusively in skeletal muscle, has been shown to increase muscle mass in several species, including humans...
January 8, 2024: Neurology and Therapy