Clonazepam repurposing in ARID1B patients through conventional RCT and N-of-1 trials: an experimental strategy for orphan disease development
通过常规随机对照试验和单例试验将氯硝西泮重新用于 ARID1B 患者:一种罕见病研发的实验策略
期刊:Journal of Medical Genetics
影响因子:3.7
doi:10.1136/jmg-2024-109951
van der Sluijs, Pleuntje J; Safai Pour, Koshar; Berends, Cécile L; Kruizinga, Matthijs D; Müller, Annelieke R; van Eeghen, Agnies M; Rodríguez-Girondo, Mar; Juachon, Maria J; Steenbeek, Duco; Cohen, Adam F; Zuiker, Rob G J A; Santen, Gijs W E