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Article

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

Orphanet journal of rare diseases - 27 Feb 2021

Forde Karina, Resta Nicoletta, Ranieri Carlotta, Rea David, Kubassova Olga, Hinton Mark, Andrews Katrina A, Semple Robert, Irvine Alan D, Dvorakova Veronika

Abstract excerpt

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 vitro efficacy. Following...

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Identifiers

PMID: 33639990
DOI: 10.1186/s13023-021-01745-0
PMCID: PMC7913425

Genes

PIK3CA
AKT1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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Clinical experience with the AKT1 inhibitor miransertib in two children with PIK3CA-related overgrowth syndrome.PMID 33639990Read ↗

Clinical Experience with the AKT1 Inhibitor Miransertib in two Children with PIK3CA-related overgrowth syndromeSemantic proximity Read ↗

In vitro efficacy of ARQ 092, an allosteric AKT inhibitor, on primary fibroblast cells derived from patients with PIK3CA-related overgrowth spectrum (PROS).Semantic proximity Read ↗

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Response to Alpelisib in Clinically Distinct Pediatric Patients With PIK3CA -related Disorders.Semantic proximity Read ↗

Alpelisib to treat CLOVES syndrome, a member of the PIK3CA-related overgrowth syndrome spectrumSemantic proximity Read ↗

Alpelisib for treatment of patients with PIK3CA-related overgrowth spectrum (PROS).Semantic proximity Read ↗

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Updates on Diagnosis and Treatment of PIK3CA-Related Overgrowth Spectrum.Semantic proximity Read ↗

PIK3CA-related overgrowth spectrum (PROS): diagnostic and testing eligibility criteria, differential diagnosis, and evaluation.Semantic proximity Read ↗

Somatic overgrowth disorders of the PI3K/AKT/mTOR pathway & therapeutic strategies.Semantic proximity Read ↗

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