Drug developed for inherited bleeding disorder shows promising trial results
Hereditary hemorrhagic telangiectasia (HHT) is the second most common inherited bleeding disorder worldwide, affecting 1 in 3,800 persons. HHT’s hallmark symptom is chronic nosebleeds, which often occur alongside other internal bleeding and vascular malformations that impact quality of life and longevity. A new study from Mass General Brigham tested the safety and efficacy of engasertib, a drug specifically designed to target the condition. This 75-participant, double-blind, placebo-controlled trial found engasertib ...