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Article

Saracatinib is an efficacious clinical candidate for fibrodysplasia ossificans progressiva

2020-10-29

Abstract excerpt

Currently, no effective therapies exist for fibrodysplasia ossificans progressiva (FOP), a rare congenital syndrome in which heterotopic bone is formed in soft tissues due to dysregulated activity of the bone morphogenetic protein (BMP) receptor kinase ALK2/ ACVR1 . From a screen of known biologically active compounds, we identified saracatinib as a potent ALK2 kinase inhibitor. In enzymatic and cell-based assays...

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Literature Corpus work
21d5dc6b-5ac3-5c0f-9451-72142ef9559f
DOI
10.1101/2020.10.29.360370
Open publication

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Saracatinib is an efficacious clinical candidate for fibrodysplasia ossificans progressivaDOI 10.1101/2020.10.29.360370
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