Alpelisib for treatment of patients with PIK3CA-related overgrowth spectrum (PROS)

PIK3CA-related overgrowth spectrum (PROS) encompasses several rare conditions resulting from activating variants in PIK3CA. Alpelisib, a PI3Kα-selective inhibitor, targets the underlying etiology of PROS, offering a novel therapeutic approach to current management strategies. This study evaluated th...

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Veröffentlicht in:	Genetics in medicine 2023-12, Vol.25 (12), p.100969, Article 100969
Hauptverfasser:	Canaud, Guillaume, Lopez Gutierrez, Juan Carlos, Irvine, Alan D., Vabres, Pierre, Hansford, Jordan R., Ankrah, Nii, Branle, Fabrice, Papadimitriou, Athanasia, Ridolfi, Antonia, O’Connell, Paul, Turner, Stuart, Adams, Denise M.
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Alpelisib Child Class I Phosphatidylinositol 3-Kinases - genetics Humans Mutation Overgrowth PIK3CA PROS Retrospective Studies Thiazoles - adverse effects
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