Comparing randomized trial designs to estimate treatment effect in rare diseases with longitudinal models: a simulation study showcased by Autosomal Recessive Cerebellar Ataxias using the SARA score - Hendrickx, Niels; Mentré, France; Hamdan, Alzahra; Karlsson, Mats O.; Hooker, Andrew C.; Traschütz, Andreas; Gagnon, Cynthia; Schüle-Freyer, Rebecca; Synofzik, Matthis; Comets, Emmanuelle; Chen, Xiaomei; Heussen, Nicole Maria; et al. (BMC medical research methodology, 2025)
Gesehen am 15.12.2025