Hereditary hemorrhagic telangiectasia (HHT), also known as Osler-Weber-Rendu syndrome, is a rare, but serious, genetic disorder that causes abnormal blood vessels to form throughout the body. These ...
Treatment with the investigational oral, allosteric, selective AKT inhibitor engasertib appeared to be safe in patients with hereditary hemorrhagic telangiectasia (HHT), and was associated with ...
Despite the considerable disease burden associated with hereditary hemorrhagic telangiectasia, no therapies are licensed for treatment. In a proof-of-concept, placebo-controlled trial, the ...