Efficacy and safety of N-acetyl-L-leucine in Niemann-Pick disease type C.
Tatiana Bremova-ErtlJens ClaassenTomas FoltanJordi Gascon-BayarriPaul GissenAndreas HahnAnhar HassanAnita HennigSimon A JonesMiriam KolnikovaKyriakos MartakisJan RaethjenUma RamaswamiReena SharmaSusanne A SchneiderPublished in: Journal of neurology (2021)
NCT03759639.
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