DMD therapy / AUM LifeTech, University of Pennsylvania - LARVOL DELTA

Novel, Clinically Relevant Composite Bioassays for Cardiac and Skeletal Muscle Protection as Conferred by Dystrophin-Mimetics (ASGCT 2024) - "We predict these measures, particularly normalizing urinary NTFT to recent cycles of eccentric contraction, to be an essential component of next generation clinical studies of DMD therapeutics...Here we present physiologically relevant assays for skeletal and cardiac muscle protection in preclinical DMD animal models. Unlike conventional measures, these assays provide the necessary resolving power to differentiate related treatment groups in blinded studies, with the potential to more confidently predict clinical outcomes of patients in clinical trials."

Clinical • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Hypertension • Muscular Dystrophy • TNNI3 • TTN