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

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. Miransert...

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Detalles Bibliográficos
Autores principales:	Forde, Karina, Resta, Nicoletta, Ranieri, Carlotta, Rea, David, Kubassova, Olga, Hinton, Mark, Andrews, Katrina A., Semple, Robert, Irvine, Alan D., Dvorakova, Veronika
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2021
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7913425/ https://www.ncbi.nlm.nih.gov/pubmed/33639990 http://dx.doi.org/10.1186/s13023-021-01745-0

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