Ben Barthel

Keywords Becker muscular dystrophy Contraction-induced injury Sevasemten EDG-5506 Fast myosin inhibitor Evidence before this study Dystrophin is a critical structural protein that links the contractile elements of the sarcomere to the basement membrane of the myofibres.

AE adverse event AUC0-tau area under the curve from time 0 to tau BMD Becker muscular dystrophy BQL below the limit of quantification BMI body mass index C24 plasma concentration at the end of the dosing interval Cmax maximum observed plasma concentration CK creatine kinase DMD Duchenne muscular dystrophy EC enteric capsule ECG electrocardiogram FVC forced vital capacity GH globally higher GL globally lower HGS hand grip strength HV healthy volunteer MAD multiple ascending dose PD...

Abstract Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by absence of the protein dystrophin, which acts as a structural link between the basal lamina and contractile machinery to stabilize muscle membranes in response to mechanical stress. In DMD, mechanical stress leads to exaggerated membrane injury and fiber breakdown, with fast fibers being the most susceptible to damage. A major contributor to this injury is muscle contraction, controlled by the motor protein myosin.