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

https://hdl.handle.net/10536/DRO/DU:30144142
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journal contribution

posted on 2024-06-05, 06:51 authored by MC Patterson, D Vecchio, E Jacklin, Larry AbelLarry Abel, H Chadha-Boreham, C Luzy, R Giorgino, JE Wraith

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

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Related Materials

1.
DOI - Is version of https://doi.org/10.1177/0883073809344222

Location

United States

Language

English

Author URL

http://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000274849500005&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=a045e4b2bb1f2b747c68c720ec8913b7

Publication classification

C1 Refereed article in a scholarly journal

Journal

Journal of Child Neurology

Volume

25

Pagination

300-305

ISSN

0883-0738

eISSN

1708-8283

Issue

3

Publisher

SAGE PUBLICATIONS INC

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Categories

Keywords

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

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