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Miglustat in Niemann-Pick disease type C patients: a review

Crossref DOI link: https://doi.org/10.1186/s13023-018-0844-0

Published Online: 2018-08-15

Published Print: 2018-12

Update policy: https://doi.org/10.1007/springer_crossmark_policy

Authors

Pineda, Mercè

Walterfang, Mark

Patterson, Marc C.
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Article History

Received: 13 February 2018

Accepted: 14 June 2018

First Online: 15 August 2018

Ethics approval and consent to participate

: Not applicable.

: Not applicable.

: MP has received consulting fees, honoraria and research grants from Actelion Pharmaceuticals Ltd. and Biomarin. MW has received travel expenses, research grant funds and consulting honoraria from Actelion Pharmaceuticals Ltd. MCP has received research grants from the National Institutes of Health [NS 65768–01], the National MS Society, and Actelion Pharmaceuticals Ltd., and has also received honoraria and consulting fees from Actelion Pharmaceuticals Ltd., Alexion, Amicus, IntraBio, Novartis, Orphazyme, Shire HGT, Stem Cells Inc., Vtesse, a stipend as Editor-in-Chief of theJournal of Child NeurologyandChild Neurology Open, and royalties from Up-To-Date.

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