Trial of N-Acetyl-l-Leucine in Niemann–Pick Disease Type C
Trial of N-Acetyl-l-Leucine in Niemann–Pick Disease Type C
About this item
Full title
Author / Creator
Bremova-Ertl, Tatiana , Ramaswami, Uma , Brands, Marion , Foltan, Tomas , Gautschi, Matthias , Gissen, Paul , Gowing, Francesca , Hahn, Andreas , Jones, Simon , Kay, Richard , Kolnikova, Miriam , Arash-Kaps, Laila , Marquardt, Thorsten , Mengel, Eugen , Park, Julien H. , Reichmannová, Stella , Schneider, Susanne A. , Sivananthan, Siyamini , Walterfang, Mark , Wibawa, Pierre , Strupp, Michael and Martakis, Kyriakos
Publisher
United States: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
United States: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
In Niemann–Pick disease type C, a rare lysosomal storage disorder, treatment with
N
-acetyl-
l
-leucine, which may ameliorate lysosomal and metabolic dysfunction, resulted in better neurologic status than placebo.
Alternative Titles
Full title
Trial of N-Acetyl-l-Leucine in Niemann–Pick Disease Type C
Authors, Artists and Contributors
Author / Creator
Ramaswami, Uma
Brands, Marion
Foltan, Tomas
Gautschi, Matthias
Gissen, Paul
Gowing, Francesca
Hahn, Andreas
Jones, Simon
Kay, Richard
Kolnikova, Miriam
Arash-Kaps, Laila
Marquardt, Thorsten
Mengel, Eugen
Park, Julien H.
Reichmannová, Stella
Schneider, Susanne A.
Sivananthan, Siyamini
Walterfang, Mark
Wibawa, Pierre
Strupp, Michael
Martakis, Kyriakos
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_2921114872
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2921114872
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa2310151
