Miglustat in adult and juvenile patients with Niemann-Pick disease type C: Long-term data from a clinical trial

Wraith, JE; Vecchio, D; Jacklin, E; Abel, Larry; Chadha-Boreham, H; Luzy, C; Giorgino, R; Patterson, MC

Miglustat in adult and juvenile patients with Niemann-Pick disease type C: Long-term data from a clinical trial

Version 2 2024-06-05, 06:51

Version 1 2020-10-16, 08:15

journal contribution

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

Miglustat in adult and juvenile patients with Niemann-Pick disease type C: Long-term data from a clinical trial

History

Journal

Molecular Genetics and Metabolism

Volume

99

Pagination

351-357

Location

United States

Publisher DOI

https://doi.org/10.1016/j.ymgme.2009.12.006

ISSN

1096-7192

eISSN

1096-7206

Language

English

Author URL

http://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000276001400003&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=a045e4b2bb1f2b747c68c720ec8913b7

Publication classification

C1 Refereed article in a scholarly journal

Issue

4

Publisher

ACADEMIC PRESS INC ELSEVIER SCIENCE

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Keywords

Science & Technology Life Sciences & Biomedicine Endocrinology & Metabolism Genetics & Heredity Medicine, Research & Experimental Research & Experimental Medicine Niemann-Pick type C Miglustat Long-term Efficacy Safety CHOLESTEROL TRAFFICKING INHIBITOR SACCADES

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Miglustat in adult and juvenile patients with Niemann-Pick disease type C: Long-term data from a clinical trial

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