Functional Outcome Measures to Optimize Drug Development in Spinal and Bulbar Muscular Atrophy: Results From a Meta-Analysis of the Global SBMA Dataset
功能性结局指标在脊髓延髓肌萎缩症药物研发优化中的应用:全球 SBMA 数据集荟萃分析结果
期刊:Neurology
影响因子:8.5
doi:10.1212/WNL.0000000000210088
Huggett, Spencer B; Tebbenkamp, Andrew T N; Rinaldi, Carlo; Jayaseelan, Dipa; Zampedri, Luca; Blasi, Lorenzo; Fortuna, Andrea; Alqahtani, Abdullah; Kokkinis, Angela; Dahlqvist, Julia; Fenu, Silvia; Cavalca, Eleonora; Bertini, Alessandro; Mariotti, Caterina; Grunseich, Christopher; Kawase, Takahiro; Kishimoto, Yoshiyuki; Yamada, Shinichiro; Katsuno, Masahisa; Fratta, Pietro; Conte, Amelia; Sabatelli, Mario; Soraru, Gianni; Vissing, John; Kang, Minsung; Park, Jin-Sung; Pareyson, Davide; Viglietta, Vissia