Long-term miglustat therapy in children with niemann-pick disease type C

Patterson, Marc C, Vecchio, Darleen, Jacklin, Elizabeth, Abel, Larry A, Chadha-Boreham, Harbajan, Luzy, Cecile, Giorgino, Ruben and Wraith, James E 2010, Long-term miglustat therapy in children with niemann-pick disease type C, Journal of Child Neurology, vol. 25, no. 3, pp. 300-305, doi: 10.1177/0883073809344222.

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Title Long-term miglustat therapy in children with niemann-pick disease type C
Author(s) Patterson, Marc C
Vecchio, Darleen
Jacklin, Elizabeth
Abel, Larry AORCID iD for Abel, Larry A orcid.org/0000-0002-2393-0644
Chadha-Boreham, Harbajan
Luzy, Cecile
Giorgino, Ruben
Wraith, James E
Journal name Journal of Child Neurology
Volume number 25
Issue number 3
Start page 300
End page 305
Total pages 6
Publisher SAGE PUBLICATIONS INC
Place of publication United States
Publication date 2010-03-01
ISSN 0883-0738
1708-8283
Keyword(s) Science & Technology
Life Sciences & Biomedicine
Clinical Neurology
Pediatrics
Neurosciences & Neurology
miglustat
Niemann-Pick disease type C
efficacy
safety
GALACTOSYL HYDROLASE DEFICIENCY
CLINICAL SPECTRUM
BRAIN-STEM
LACTOSYLCERAMIDE
CHOLESTEROL
MUTATIONS
HE-1
NPC1
Language eng
DOI 10.1177/0883073809344222
Indigenous content off
Field of Research 1103 Clinical Sciences
1109 Neurosciences
1702 Cognitive Sciences
HERDC Research category C1 Refereed article in a scholarly journal
Persistent URL http://hdl.handle.net/10536/DRO/DU:30144142

Document type: Journal Article
Collections: Faculty of Health
School of Medicine
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