Safety and efficacy of low-dose sirolimus in the PIK3CA-related overgrowth spectrum
低剂量西罗莫司治疗PIK3CA相关过度生长谱的安全性和有效性
期刊:Genetics in Medicine
影响因子:6.2
doi:10.1038/s41436-018-0297-9
Parker, Victoria E R; Keppler-Noreuil, Kim M; Faivre, Laurence; Luu, Maxime; Oden, Neal L; De Silva, Leena; Sapp, Julie C; Andrews, Katrina; Bardou, Marc; Chen, Kong Y; Darling, Thomas N; Gautier, Elodie; Goldspiel, Barry R; Hadj-Rabia, Smail; Harris, Julie; Kounidas, Georgios; Kumar, Parag; Lindhurst, Marjorie J; Loffroy, Romaric; Martin, Ludovic; Phan, Alice; Rother, Kristina I; Widemann, Brigitte C; Wolters, Pamela L; Coubes, Christine; Pinson, Lucile; Willems, Marjolaine; Vincent-Delorme, Catherine; Vabres, Pierre; Semple, Robert K; Biesecker, Leslie G