Ataluren improves hematopoietic and pancreatic disorders in Shwachman-Diamond syndrome patients: a compassionate program case-series.
Ataluren 改善了施瓦赫曼-戴蒙德综合征患者的造血和胰腺疾病:一项关怀计划病例系列
期刊:Nature Communications
影响因子:15.7
doi:10.1038/s41467-025-63137-3
Bezzerri Valentino, Pegoraro Anna, Hristodor Anca Manuela, Crane Genevieve M, Meneghelli Ilaria, Brignole Cecilia, Boni Christian, Baldisseri Elena, Vella Antonio, Menichetti Giacomo, Valli Roberto, Porta Giovanni, D'Amico Giovanna, Tecchio Cristina, Parisi Alice, Lippi Giuseppe, Mercuri Angela, Cesaro Simone, Corey Seth J, Cipolli Marco