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BSH 2023 | Developing novel treatment approaches for hereditary hemorrhagic telangiectasia

Hanny Al-Samkari, MD, Massachusetts General Hospital, Boston, MA, discusses the current lack of approved treatments for patients with hereditary hemorrhagic telangiectasia (HHT). Dr Al-Samkari provides an overview of the pathophysiology and primary symptoms of HHT and further describes two categories of therapeutics under development: drugs repurposed from cancer treatment, such as bevacizumab, thalidomide, and pomalidomide, and drugs being specifically developed for HHT, such as the allosteric AKT inhibitor VAD044. Of these agents, Dr Al-Samkari notes that bevacizumab, pomalidomide, and VAD044 are the subjects of ongoing clinical trials. This interview took place at the 63rd Annual Scientific Meeting of the British Society for Haematology (BSH) 2023, held in Birmingham, UK.

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Disclosures

Research funding to institution (Agios, Amgen, Novartis, Vaderis, Sobi); Consultancy (Agios, Novartis, Moderna, Rigel, argenx, Pharmacosmos, Sobi, Forma)