A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia
一项旨在研究新型疗法乙酰-L-亮氨酸治疗三种极其罕见的神经退行性疾病（尼曼-匹克C型、GM2神经节苷脂沉积症和毛细血管扩张性共济失调）的主方案。
期刊:Trials
影响因子:2
doi:10.1186/s13063-020-05009-3
Fields, T; Patterson, M; Bremova-Ertl, T; Belcher, G; Billington, I; Churchill, G C; Davis, W; Evans, W; Flint, S; Galione, A; Granzer, U; Greenfield, J; Karl, R; Kay, R; Lewi, D; Mathieson, T; Meyer, T; Pangonis, D; Platt, F M; Tsang, L; Verburg, C; Factor, M; Strupp, M
