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Recent Advances in ALK2 Inhibitors.


ABSTRACT: Activin receptor-like kinase-2 (ALK2) is a type I bone morphogenetic protein (BMP) receptor which has a role in biological processes that control the development of bone, heart, brain, and other tissue. Gain of function mutations in ALK2 have been identified in fibrodysplasia ossificans progressiva (FOP) and the childhood brain tumor, diffuse intrinsic pontine glioma (DIPG), which has given focus to the development of ALK2 inhibitors as targeted treatments. This review covers the structural features of ALK2 inhibitors which contribute to their ALK2 potency and selectivity, and the pharmacokinetic or in vivo efficacy data available to demonstrate their suitability for treating a peripheral or CNS disease.

SUBMITTER: Rooney L 

PROVIDER: S-EPMC8374899 | biostudies-literature |

REPOSITORIES: biostudies-literature

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