Publication
Methodological challenges in outcomes research for early-trials for implementation of new therapies in neuropediatric rare diseases
Maria T. Acosta, Silvia Zaragoza Domingo, Celso Arango, Kim I. Bishop, Joan Busner, Georg Dorffner, Inés del Cerro, John Carlos Diaz, Edna Gruenblatt, Sabine M. Hölter, Brian Harel, Vanitha Krishna, Marta Mas, Carmen Moreno, Stefano Pallanti, Carme Plasencia, Sarah Kittel-Schneider, Daniella Tinoco, Monica Vance, Manpreet K. Singh, Simona Giorgi, José Ángel Aibar, Antonella Santuccione Chadha, Estibaliz Arce Cirauqui
Orphanet Journal of Rare Diseases, October 2025, Springer Science + Business Media
DOI: 10.1186/s13023-025-03814-0