Safety and efficacy of droxidopa for dysautonomia in adults with Menkes disease and occipital horn syndrome in the USA: a randomised phase 1/2a crossover trial
在美国,一项随机1/2a期交叉试验评估了屈昔多巴治疗成人门克斯病和枕角综合征患者自主神经功能障碍的安全性和有效性。
期刊:EClinicalMedicine
影响因子:10
doi:10.1016/j.eclinm.2026.103898
Kaler, Maryann M; Brock, Guy; Jeanty, Christopher J; Iammarino, Megan A; Hampton, Kimberly A; Pham, Minh T; Sabo, Brenna T; Lowes, Linda P; Sullivan, Patricia; Goldstein, David S; Kaler, Stephen G