Article

Repurposing alpelisib, an anti-cancer drug, for the treatment of severe TIE2-mutated venous malformations: preliminary pharmacokinetics and pharmacodynamic data

2022-03-15

Abstract excerpt

Extensive venous malformations (VM) involving limbs severely impact quality of life, mostly due to chronic pain and functional limitations. Patients can also display coagulopathy with associated risks of life-threatening thromboembolism and bleeding. Current pharmacological VM treatments (e.g. sirolimus) are not universally effective as 10% of patients present intractable debilitating and/or critical disease. Nove...

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Literature Corpus work: 466fa58d-c767-581c-8d3a-7f323e0f9ec1
DOI: 10.21203/rs.3.rs-1440565/v1

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