Vascular malformations in 2023: Sirolimus confirms its efficacy and allows treatment in utero

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Emmanuel Seront MD PhD1, An Van Damme MD PhD2, Julien Coulie MD3, Valérie Dekeuleneer MD3, Miikka Vikkula MD PhD4, Laurence M Boon, MD PhD3,4 Published in the journal : February 2024 Category : Chirurgie vasculaire

Summary :

The mTOR pathway plays an essential role in the development of vascular malformations. A recently published VASE study confirms the efficacy of sirolimus, an mTOR inhibitor, in vascular malformations, with an efficacy rate of 85% and good overall safety.

Keywords

mTOR, sirolimus, TIE2, PIK3CA, vascular malformation, in utero

French website for full article

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