Clinical experience with the AKT1 inhibitor miransertib in two children with PIK3CA-related overgrowth syndrome

Forde, K; Resta, N; Ranieri, C; Rea, D; Kubassova, O; Hinton, M; Andrews, KA; Semple, R; Irvine, AD; Dvorakova, V

Irvine, AD (corresponding author), Childrens Hlth Ireland Crumlin, Dermatol, Dublin 12, Ireland.; Irvine, AD (corresponding author), Trinity Coll Dublin, Clin Med, Dublin, Ireland.

ORPHANET JOURNAL OF RARE DISEASES, 2021; 16 (1):

Abstract

BackgroundPIK3CA-related overgrowth spectrum (PROS) refers to a group of rare disorders, caused by somatic activating mutations in PIK3CA, resulting i......

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