Methodological challenges in outcomes research for early-trials for implementation of new therapies in neuropediatric rare diseases
神经儿科罕见病新疗法早期试验结果研究的方法学挑战
期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-025-03814-0
Acosta, Maria T; Zaragoza Domingo, Silvia; Arango, Celso; Bishop, Kim I; Busner, Joan; Dorffner, Georg; Cerro, Inés Del; Diaz, John Carlos; Gruenblatt, Edna; Hölter, Sabine M; Harel, Brian; Krishna, Vanitha; Mas, Marta; Moreno, Carmen; Pallanti, Stefano; Plasencia, Carme; Kittel-Schneider, Sarah; Tinoco, Daniella; Vance, Monica; Singh, Manpreet K; Giorgi, Simona; Aibar, José Ángel; Chadha, Antonella Santuccione; Cirauqui, Estibaliz Arce