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

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 m...

Source: Orphanet Journal of Rare Diseases - February 27, 2021 Category: Internal Medicine Authors: Karina Forde, Nicoletta Resta, Carlotta Ranieri, David Rea, Olga Kubassova, Mark Hinton, Katrina A. Andrews, Robert Semple, Alan D. Irvine and Veronika Dvorakova Tags: Research Source Type: research