N-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study

被引：0
作者：
T Fields
T M. Bremova
I Billington
GC Churchill
W Evans
C Fields
A Galione
R Kay
T Mathieson
K Martakis
M Patterson
F Platt
M Factor
M Strupp
机构：
[1] IntraBio Ltd,Department of Neurology
[2] Begbroke Science Park,Department of Pharmacology
[3] Inselspital,Primary Care Stratified Medicine (PRISM), Division of Primary Care
[4] University Hospital Bern,Department of Pediatric Neurology
[5] and University of Bern,Department of Neurology and German Center for Vertigo and Balance Disorders
[6] University of Oxford,undefined
[7] Niemann-Pick UK,undefined
[8] University of Nottingham,undefined
[9] RK Statistics,undefined
[10] Brook House,undefined
[11] University Children’s Hospital (UKGM) and Medical Faculty,undefined
[12] Justus Liebig University of Giessen,undefined
[13] Mayo Clinic,undefined
[14] Ludwig Maximilians University,undefined
来源：
Trials | / 24卷
关键词：
Niemann-Pick type C (NPC); N-acetyl-L-leucine; Pharmaceutical intervention; Symptomatic treatment; Randomized controlled trial; Cerebellar ataxia; Lysosomal storage disease;
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