Output details
11 - Computer Science and Informatics
University of Plymouth
Article title
A randomized controlled trial of miglustat in Gaucher disease type 3.
Type
D - Journal article
Title of journal
Annals of Neurology
Article number
-
Volume number
64
Issue number
5
First page of article
514
ISSN of journal
1531-8249
Year of publication
2008
URL
-
Number of additional authors
10
Additional information
<24> This paper reports the results of phase 2 translational research into an international randomised placebo controlled trial to test the efficacy of miglustat in slowing neural degeneration in the lysosomal storage disease Gaucher type 3, (funded by Oxford Glycosciences). Harris was the investigator, recording and analyzing data, for the UK arm of the study. Abnormal slowing of saccades is an early sign of brainstem degeneration in this disease (Harris et al. Neuropediatrics, 1999). This was the first paediatric RCT to use saccades as the principle outcome measure.
Interdisciplinary
Yes
Cross-referral requested
4 - Psychology, Psychiatry and Neuroscience
Research group
None
Citation count
54
Proposed double-weighted
No
Double-weighted statement
-
Reserve for a double-weighted output
No
Non-English
No
English abstract
-
