In a 36-week placebo-controlled phase 2 trial in patients with non-alcoholic steatohepatitis (NASH), treatment with mgl-3196 (resmetirom) significantly reduces atherogenic lipoprotein particles
Novel cardiotropic AAV variant C102 vectors show superior gene delivery & reduced immunogenicity in non-human primates, transduction of human cardiomyocytes, & correction of Fabry disease phenotype