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Search Results - Machielse, Bernardus N
Search Results - Machielse, Bernardus N
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Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial
by
Ory, Daniel S
,
Ottinger, Elizabeth A
,
Farhat, Nicole Yanjanin
,
King, Kelly A
,
Jiang, Xuntian
,
Weissfeld, Lisa
,
Berry-Kravis, Elizabeth
,
Davidson, Cristin D
,
Bianconi, Simona
,
Keener, Lee Ann
,
Rao, Ravichandran
,
Soldatos, Ariane
,
Sidhu, Rohini
,
Walters, Kimberly A
,
Xu, Xin
,
Thurm, Audrey
,
Solomon, Beth
,
Pavan, William J
,
Machielse, Bernardus N
,
Kao, Mark
,
Silber, Steven A
,
McKew, John C
,
Brewer, Carmen C
,
Vite, Charles H
,
Walkley, Steven U
,
Austin, Christopher P
,
Porter, Forbes D
Published in
The Lancet (British edition)
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VTS-270 for the treatment of Niemann-Pick disease type C
by
Machielse, Bernardus N
,
Porter, Forbes D
,
Yergey, Alfred L
,
Berry-Kravis, Elizabeth M
,
Darling, Allan
,
Rao, Ravi
Published in
Molecular genetics and metabolism
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Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick Disease, type C1: an ad-hoc analysis of a non-randomized, open-label, phase 1/...
by
Ory, Daniel S.
,
Ottinger, Elizabeth A.
,
Farhat, Nicole Yanjanin
,
King, Kelly A.
,
Jiang, Xuntian
,
Weissfeld, Lisa
,
Berry-Kravis, Elizabeth
,
Davidson, Cristin D.
,
Bianconi, Simona
,
Keener, Lee Ann
,
Rao, Ravichandran
,
Soldatos, Ariane
,
Sidhu, Rohini
,
Walters, Kimberly A.
,
Xu, Xin
,
Thurm, Audrey
,
Solomon, Beth
,
Pavan, William J.
,
Machielse Drs, Bernardus N.
,
Kao, Mark
,
Silber, Steven A.
,
McKew, John C.
,
Brewer, Carmen C.
,
Vite, Charles H.
,
Walkley, Steven U.
,
Austin, Christopher P.
,
Porter, Forbes D.
Published in
The Lancet (British edition)
Get full text
Article
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Journal Title
The Lancet
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Lancet
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Molecular Genetics And Metabolism
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Subjects
2-Hydroxypropyl-Beta-Cyclodextrin - Administration & Dosage
1 results
1
2-Hydroxypropyl-Beta-Cyclodextrin - Adverse Effects
1 results
1
Adolescent
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Age
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Animal Models
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Bioindicators
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Biomarkers
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Biomarkers - Blood
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Biomarkers - Cerebrospinal Fluid
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Brain Diseases
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Calbindins - Cerebrospinal Fluid
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Cerebellum
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Cerebrospinal Fluid
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Child
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Child, Preschool
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Childrens Health
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Cholesterol
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Clinical Trials
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Cognition
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Cyclodextrins
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Sciencedirect (Online Service)
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Elsevier
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Ebscohost Business Source Ultimate
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Science Citation Index Expanded (Web Of Science)
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