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

Internet

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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