Miglustat as a Treatment for Adults with Tangier Disease Neuropathy: The MUSTANG N-of-1 Trial with 21 months Clinical Observation
米格鲁司他治疗成人唐吉尔病神经病变:MUSTANG N-of-1 试验及 21 个月临床观察
期刊:Neurology and Therapy
影响因子:4.8
doi:10.1007/s40120-025-00843-6
Cook, Andrew; Stuart, Beth; Ochoa-Ferraro, Antonio; Condon, Nicola; Lawrence, Megan; Webley, Fran; Wallom, Kerri-Lee; Forbes, Claire; Veeranna, Vishy; Wanninayake, Subadra; Oliver, Tom; Davies, Nicholas; Dawson, Charlotte; Platt, Frances; Geberhiwot, Tarekegn