Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

Patterson, MC; Vecchio, D; Prady, H; Abel, Larry; Wraith, JE

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Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

Version 2 2024-06-05, 06:51

Version 1 2020-10-16, 08:17

journal contribution

posted on 2024-06-05, 06:51 authored by MC Patterson, D Vecchio, H Prady, Larry AbelLarry Abel, JE Wraith

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

History

Journal

Lancet Neurology

Volume

6

Pagination

765-772

Location

England

Publisher DOI

https://doi.org/10.1016/S1474-4422(07)70194-1

ISSN

1474-4422

eISSN

1474-4465

Language

eng

Author URL

https://www.ncbi.nlm.nih.gov/pubmed/17689147

Publication classification

C1 Refereed article in a scholarly journal

Issue

9

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Keywords

1-Deoxynojirimycin Adolescent Adult Age Factors Analysis of Variance Child Child, Preschool Cohort Studies Deglutition Double-Blind Method Drug Administration Schedule Enzyme Inhibitors Female Humans Male Niemann-Pick Disease, Type C Retrospective Studies Saccades Severity of Illness Index

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File(s) under permanent embargo

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

History

Journal

Volume

Pagination

Location

Publisher DOI

ISSN

eISSN

Language

Author URL

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Issue

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Keywords

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