Efficacy and Safety of N-Acetyl-l-Leucine in Children and Adults With GM2 Gangliosidoses
N-乙酰-L-亮氨酸治疗儿童和成人GM2神经节苷脂病的疗效和安全性
期刊:Neurology
影响因子:8.5
doi:10.1212/WNL.0000000000201660
Martakis, Kyriakos; Claassen, Jens; Gascon-Bayari, Jordi; Goldschagg, Nicolina; Hahn, Andreas; Hassan, Anhar; Hennig, Anita; Jones, Simon; Kay, Richard; Lau, Heather; Perlman, Susan; Sharma, Reena; Schneider, Susanne; Bremova-Ertl, Tatiana