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...

Olles dieđut

Bibliográfalaš dieđut

Váldodahkkit:	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
Materiálatiipa:	Artigo
Giella:	Inglês
Almmustuhtton:	BMC 2025-07-01
Ráidu:	Orphanet Journal of Rare Diseases
Fáttát:	Miransertib AKT PIK3CA Proteus syndrome PIK3CA-related overgrowth spectrum
Liŋkkat:	https://doi.org/10.1186/s13023-025-03831-z
Fáddágilkorat:	Lasit fáddágilkoriid Eai fáddágilkorat, Lasit vuosttaš fáddágilkora!