Fast skeletal muscle troponin activator in the dy2J muscular dystrophy model.

摘要: Introduction: Tirasemtiv is a novel small molecule activator of the fast skeletal muscle troponin complex that pro- duces sensitization sarcomere to calcium. currently in Phase II clinical trials for neuromuscular disease. Methods: We conducted blinded, randomized, placebo-con- trolled preclinical study effect tirasemtiv on forearm grip strength, endurance, respiratory physiology, and pathology adequate sample sizes Lama2 dy-2J mouse model congenital muscular dystrophy. Results: Mice receiving high dose had significantly higher fiber cross-sectional area response CO2 stimula- tion at 16 weeks than mice low or placebo. There were no changes pathology, serum creatine kinase, respiration following long-term treat- ment. Conclusions: conclude influences structure this dystrophy but does not impact function, as evaluated study. Muscle Nerve 48: 279-285, 2013 Merosin-deficient (MDC1A) an autosomal recessive disorder char-