Safety findings from the phase 1/2 MOSAIC study of miransertib for patients with PIK3CA-related overgrowth spectrum or Proteus syndrome

Abstract Background PIK3CA-related overgrowth spectrum (PROS) and Proteus syndrome are associated with mosaic tissue overgrowth of varying severity that commonly presents in childhood. The multicenter, open-label, phase 1/2 MOSAIC study (NCT03094832) was designed to evaluate the clinical efficacy an...

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Principais autores:	Whitney Eng, Ionela Iacobas, Jonathan Perkins, Giuseppe Zampino, Chiara Leoni, Paola Sabrina Buonuomo, Alessandra Simonetti, Himanshu Goel, Michael Briones, Mo Huang, Gregory Goldmacher, Danny Liaw, Adrienne Hammill
Format:	Artigo
Jezik:	Inglês
Izdano:	BMC 2025-07-01
Serija:	Orphanet Journal of Rare Diseases
Teme:	Miransertib AKT PIK3CA Proteus syndrome PIK3CA-related overgrowth spectrum
Online dostop:	https://doi.org/10.1186/s13023-025-03831-z
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