(Q38627151)

English

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial

scientific article published on 10 August 2017

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scholarly article

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Europe PubMed Central

PubMed publication ID

4 September 2017

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial (English)

1 reference

Europe PubMed Central

PubMed publication ID

4 September 2017

phase I clinical trial

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phase II clinical trial

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Niemann-Pick disease

1 reference

based on heuristic

inferred from title

Niemann-Pick disease, type C1

1 reference

based on heuristic

inferred from title

object named as

Rohini Sidhu

13

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William J Pavan

object named as

William J Pavan

18

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Cristin Davidson

object named as

Cristin D Davidson

8

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Christopher P Austin

26

object named as

Christopher P Austin

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Europe PubMed Central

PubMed publication ID

4 September 2017

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Daniel S Ory

1

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Europe PubMed Central

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4 September 2017

Elizabeth A Ottinger

2

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Europe PubMed Central

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4 September 2017

Nicole Yanjanin Farhat

3

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4 September 2017

Kelly A King

4

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4 September 2017

Xuntian Jiang

5

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4 September 2017

Lisa Weissfeld

6

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4 September 2017

Elizabeth Berry-Kravis

7

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4 September 2017

Simona Bianconi

9

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4 September 2017

Lee Ann Keener

10

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4 September 2017

Ravichandran Rao

11

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4 September 2017

Ariane Soldatos

12

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4 September 2017

Kimberly A Walters

14

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4 September 2017

Xin Xu

15

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4 September 2017

Audrey Thurm

16

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4 September 2017

Beth Solomon

17

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4 September 2017

Bernardus N Machielse

19

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4 September 2017

Mark Kao

20

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4 September 2017

Steven A Silber

21

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Europe PubMed Central

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4 September 2017

John C McKew

22

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Europe PubMed Central

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4 September 2017

Carmen C Brewer

23

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Europe PubMed Central

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4 September 2017

Charles H Vite

24

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Europe PubMed Central

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4 September 2017

Steven U Walkley

25

1 reference

Europe PubMed Central

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4 September 2017

Forbes D Porter

27

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4 September 2017

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publication date

10 August 2017

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Europe PubMed Central

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4 September 2017

1 reference

Europe PubMed Central

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4 September 2017

Effects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann-Pick Type C disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Cerebrospinal Fluid Calbindin D Concentration as a Biomarker of Cerebellar Disease Progression in Niemann-Pick Type C1 Disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann-Pick C1.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Cholesterol homeostatic responses provide biomarkers for monitoring treatment for the neurodegenerative disease Niemann-Pick C1 (NPC1)

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Phosphorylated tau-Aβ42 ratio as a continuous trait for biomarker discovery for early-stage Alzheimer's disease in multiplex immunoassay panels of cerebrospinal fluid

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Auditory phenotype of Niemann-Pick disease, type C1.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Heart fatty acid binding protein and Aβ-associated Alzheimer's neurodegeneration

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Niemann-Pick disease type C clinical database: cognitive and coordination deficits are early disease indicators

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Cyclodextrin alleviates neuronal storage of cholesterol in Niemann-Pick C disease without evidence of detectable blood-brain barrier permeability

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Hearing loss and hair cell death in mice given the cholesterol-chelating agent hydroxypropyl-β-cyclodextrin

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Effects of cyclodextrin in two patients with Niemann-Pick Type C disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Quantitative proteomic analysis of Niemann-Pick disease, type C1 cerebellum identifies protein biomarkers and provides pathological insight

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Defining natural history: assessment of the ability of college students to aid in characterizing clinical progression of Niemann-Pick disease, type C

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Niemann-Pick disease type C

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

2-hydroxypropyl-beta-cyclodextrin raises hearing threshold in normal cats and in cats with Niemann-Pick type C disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Long-term miglustat therapy in children with Niemann-Pick disease type C.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Linear clinical progression, independent of age of onset, in Niemann-Pick disease, type C

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1-/- mouse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

NPC-db, a Niemann-Pick type C disease gene variation database

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Genetic variations and treatments that affect the lifespan of the NPC1 mouse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

The natural history of Niemann-Pick disease type C in the UK.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Niemann-Pick type C disease involves disrupted neurosteroidogenesis and responds to allopregnanolone

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Metastatic hepatocellular carcinoma in a patient with niemann-pick disease, type C.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Niemann-Pick disease: a review of eighteen patients.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Cholesterol homeostasis in human brain: turnover of 24S-hydroxycholesterol and evidence for a cerebral origin of most of this oxysterol in the circulation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Cerebrospinal fluid 28-kDa calbindin-D as a possible marker for Purkinje cell damage

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Niemann-Pick disease type C: Diagnosis and outcome in children, with particular reference to liver disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Uses and abuses of hearing loss classification.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Progression of neurovisceral storage disease with supranuclear ophthalmoplegia following orthotopic liver transplantation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Audiologic profile in Niemann-Pick C.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6176479

3 December 2018

Hepatocellular carcinoma in a child with Niemann-Pick disease: imaging findings

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2817%2931465-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Lung toxicity of hydroxypropyl-β-cyclodextrin infusion

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2817%2931465-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Use of 2 hydroxypropyl-beta-cyclodextrin therapy in two adult Niemann Pick Type C patients

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2817%2931465-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Identifiers

10.1016/S0140-6736(17)31465-4

1 reference

Europe PubMed Central

PubMed publication ID

4 September 2017

release_envolcusifa7xdf5jf2gsigfqq

1 reference

https://api.fatcat.wiki/v0/release/envolcusifa7xdf5jf2gsigfqq

24 November 2022

based on heuristic

mapped directly with Wikidata item

PMC publication ID

0 references

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

4 September 2017

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