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A randomized, double-blind placebo-controlled proof-of-concept trial of engasertib (AKT inhibitor) in hereditary hemorrhagic telangiectasia showed modest reductions in epistaxis frequency and duration versus placebo and a tolerable safety p
In a randomized, double‑blind, placebo‑controlled proof‑of‑concept trial, oral engasertib reduced epistaxis frequency and duration in hereditary hemorrhagic telangiectasia (HHT) with a safety profile similar to placebo except for reversible
Recent research identifies PIEZO1 overexpression as a key driver of arteriovenous malformations in type 2 hereditary hemorrhagic telangiectasia, revealing a novel pathway for targeted therapy.