Oral splicing modulator branaplam in Huntington's disease: a phase 2 randomized controlled trial
口服剪接调节剂布拉那普拉姆治疗亨廷顿病:一项 II 期随机对照试验
期刊:Nature Medicine
影响因子:50
doi:10.1038/s41591-025-04117-4
Borowsky, Beth; Ramos, Harry; Caputo, Angelika; Hartmann, Andreas; Faller, Thomas; Peters, Thomas; Sui, Yihan; Liu, Fonda; Meadowcroft, Mark; David, Olivier J; Laisney, Marc; Kinhikar, Arvind; Marder, Karen S; Tabrizi, Sarah J; Landwehrmeyer, G Bernhard; Leavitt, Blair R