Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale
一项为期 12 个月的双盲随机试验评估了阿瑞莫克洛治疗尼曼-匹克病 C 型 (NPC) 的疗效：提出了一种重新评分的 4 域 NPC 临床严重程度量表。
期刊:Molecular Genetics and Metabolism Reports
影响因子:1.9
doi:10.1016/j.ymgmr.2025.101233
Mengel, Eugen; Patterson, Marc C; Da Riol, Rosalia M; Del Toro, Mireia; Deodato, Federica; Gautschi, Matthias; Grunewald, Stephanie; Grønborg, Sabine Weller; Harmatz, Paul; Hennermann, Julia B; Héron, Bénédicte; Maier, Esther M; Roubertie, Agathe; Santra, Saikat; Tylki-Szymanska, Anna; LaGorio, Lisa; Berry-Kravis, Elizabeth; Porter, Forbes D; Solomon, Beth; Himmelstrup, Louise; Mickle, Travis; Guenther, Sven; Dali, Christine Í
