Article

Clinical Experience with the AKT1 Inhibitor Miransertib in two Children with PIK3CA-related overgrowth syndrome

2020-05-29

Abstract excerpt

<title>Abstract</title> <p>Background PIK3CA-related overgrowth spectrum (PROS) refers to a group of rare disorders, caused by somatic activating mutations in PIK3CA, resulting in abnormal PI3K-AKT-mTOR pathway signalling. Significant associated morbidity is frequently observed, and approved treatments are lacking. Miransertib (ARQ 092) is a novel, orally available, selective pan-AKT inhibitor with proven in vitr...

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Literature Corpus work: c70dd493-56d8-58c8-9c39-33bfd3c8fb04
DOI: 10.21203/rs.3.rs-31231/v1

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