Disease-Modifying, Neuroprotective Effect of N-Acetyl-l-Leucine in Adult and Pediatric Patients With Niemann-Pick Disease Type C
N-乙酰-L-亮氨酸对成人和儿童尼曼-匹克病C型患者的疾病改善和神经保护作用
期刊:Neurology
影响因子:8.5
doi:10.1212/WNL.0000000000213589
Patterson, Marc C; Ramaswami, Uma; Donald, Aimee; Foltan, Tomas; Gautschi, Matthias; Gissen, Paul; Hahn, Andreas; Jones, Simon A; Kay, Richard; Kolniková, Miriam; Park, Julien; Reichmannová, Stella; Walterfang, Mark; Wibawa, Pierre; Rohrbach, Marianne; Martakis, Kyriakos; Bremova-Ertl, Tatiana