Disease-Modifying, Neuroprotective Effect of N-Acetyl-l-Leucine in Adult and Pediatric Patients With Niemann-Pick Disease Type C.
MC Patterson , U Ramaswami , A Donald , T Foltan , M Gautschi , P Gissen , A Hahn , SA Jones , R Kay , M Kolniková , J Park , S Reichmannová , M Walterfang , P Wibawa , M Rohrbach , K Martakis , T Bremova-Ertl
Research article (journal) | Peer reviewedDetails about the publication
Journal: Neurology
Volume: 105
Issue: 1
Status: Published
Release year: 2025 (06/01/2026)
Link to the full text: https://doi.org/10.1212/WNL.0000000000213589
Keywords: niemann-pick disease type C; lysosomal storage disease
Authors from the University of Münster
| Park, Julien Heinrich | University Children's Hospital - Department for General Paediatrics |