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Article

KRAS-driven model of Gorham-Stout disease effectively treated with trametinib

22 Jun 2021

Abstract excerpt

Gorham-Stout disease (GSD) is a sporadically occurring lymphatic disorder. Patients with GSD develop ectopic lymphatics in bone, gradually lose bone, and can have life-threatening complications, such as chylothorax. The etiology of GSD is poorly understood, and current treatments for this disease are inadequate for most patients. To explore the pathogenesis of GSD, we performed targeted high-throughput sequencing...

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PMID: 34156985
DOI: 10.1172/jci.insight.149831

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