Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis
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Title
Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis
Authors
Keywords
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Journal
Orphanet Journal of Rare Diseases
Volume 14, Issue 1, Pages -
Publisher
Springer Science and Business Media LLC
Online
2019-10-21
DOI
10.1186/s13023-019-1207-1
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