Drug developed for inherited bleeding disorder shows promising trial results
Peer-Reviewed Publication
Updates every hour. Last Updated: 30-Nov-2025 21:11 ET (1-Dec-2025 02:11 GMT/UTC)
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 was safe and decreased nosebleed frequency and duration. Their results are published in New England Journal of Medicine.
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