(Q27006029)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24283970%20AND%20SRC:MED&resulttype=core&format=json

1 March 2020

title

Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease (English)

2 references

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1 March 2020

1 reference

Nuria Carrillo-Carrasco

3

1 reference

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1 March 2020

Wei Zheng

14

1 reference

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1 March 2020

William J Pavan

17

1 reference

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1 March 2020

John Heiss

18

1 reference

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1 March 2020

24

Christopher P Austin

1 reference

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1 March 2020

13

Juan Marugan

1 reference

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1 March 2020

12

Seameen J Dehdashti

1 reference

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1 March 2020

23

Forbes D Porter

1 reference

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1 March 2020

21

Daniel S Ory

1 reference

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1 March 2020

20

Steven U Walkley

1 reference

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1 March 2020

25

John C McKew

1 reference

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1 March 2020

author name string

Elizabeth A Ottinger

1

1 reference

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1 March 2020

Mark L Kao

2

1 reference

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1 March 2020

Nicole Yanjanin

4

1 reference

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1 March 2020

Roopa Kanakatti Shankar

5

1 reference

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1 March 2020

Marjo Janssen

6

1 reference

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1 March 2020

Marcus Brewster

7

1 reference

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1 March 2020

Ilona Scott

8

1 reference

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1 March 2020

Xin Xu

9

1 reference

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1 March 2020

Jim Cradock

10

1 reference

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1 March 2020

Pramod Terse

11

1 reference

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1 March 2020

Lili Portilla

15

1 reference

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1 March 2020

Alan Hubbs

16

1 reference

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1 March 2020

Charles H Vite

19

1 reference

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1 March 2020

Steven A Silber

22

1 reference

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1 March 2020

language of work or name

1 reference

1 January 2014

1 reference

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Current Topics in Medicinal Chemistry

1 March 2020

published in

2 references

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1 March 2020

volume

14

2 references

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1 March 2020

issue

3

2 references

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1 March 2020

page(s)

330-339

1 reference

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Translational research and the evolving landscape for biomedical innovation

1 March 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Niemann-Pick disease type C

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

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

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

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

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Niemann-Pick C1 disease gene: homology to mediators of cholesterol homeostasis

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Cyclodextrin induces calcium-dependent lysosomal exocytosis

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

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

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Neuroprotection by cyclodextrin in cell and mouse models of Alzheimer disease

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Microarray expression analysis and identification of serum biomarkers for Niemann-Pick disease, type C1.

28 September 2017

1 reference

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Recommendations for the diagnosis and management of Niemann-Pick disease type C: an update

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Normalization of cholesterol homeostasis by 2-hydroxypropyl-β-cyclodextrin in neurons and glia from Niemann-Pick C1 (NPC1)-deficient mice

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

A sensitive and specific LC-MS/MS method for rapid diagnosis of Niemann-Pick C1 disease from human plasma

28 September 2017

1 reference

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Quantitative role of LAL, NPC2, and NPC1 in lysosomal cholesterol processing defined by genetic and pharmacological manipulations

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Accelerating orphan drug development

28 September 2017

1 reference

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Cholesterol oxidation products are sensitive and specific blood-based biomarkers for Niemann-Pick C1 disease

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Pharmaceutical applications of cyclodextrins: basic science and product development

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

A call for sharing: adapting pharmaceutical research to new realities.

28 September 2017

1 reference

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Miglustat in adult and juvenile patients with Niemann-Pick disease type C: long-term data from a clinical trial

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Cyclodextrin overcomes the transport defect in nearly every organ of NPC1 mice leading to excretion of sequestered cholesterol as bile acid

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Effects of cyclodextrins on drug delivery through biological membranes.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Cyclodextrins as pharmaceutical solubilizers.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

The adult form of Niemann-Pick disease type C.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Cyclodextrins in drug delivery.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

2-Hydroxypropyl-beta-cyclodextrin (HP-beta-CD): a toxicology review

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Cyclodextrin-based pharmaceutics: past, present and future

28 September 2017

1 reference

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Niemann-Pick disease type C.

28 September 2017

1 reference

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Niemann-Pick type C: a disorder of cellular cholesterol trafficking

28 September 2017

1 reference

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Apolipoprotein A-I, cyclodextrins and liposomes as potential drugs for the reversal of atherosclerosis. A review.

28 September 2017

1 reference

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Pharmaceutical applications of cyclodextrins. III. Toxicological issues and safety evaluation

28 September 2017

1 reference

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Pharmaceutical applications of cyclodextrins. 2. In vivo drug delivery

28 September 2017

1 reference

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Pharmaceutical applications of cyclodextrins. 1. Drug solubilization and stabilization

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

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

28 September 2017

1 reference

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Unesterified cholesterol accumulation in late endosomes/lysosomes causes neurodegeneration and is prevented by driving cholesterol export from this compartment

31 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Weekly cyclodextrin administration normalizes cholesterol metabolism in nearly every organ of the Niemann-Pick type C1 mouse and markedly prolongs life.

31 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Murine model of Niemann-Pick C disease: mutation in a cholesterol homeostasis gene.

31 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Cellular cholesterol efflux mediated by cyclodextrins

31 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.

31 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

The effect of parenterally administered cyclodextrins on the pharmacokinetics of coadministered drugs

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

What the Orphan Drug Act has done lately for children with rare diseases: a 10-year analysis

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Does orphan drug legislation really answer the needs of patients?

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Dysfunction of the cholesterol biosynthetic pathway in Huntington's disease.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Cyclodextrins differentially mobilize free and esterified cholesterol from primary human foam cell macrophages

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Cyclodextrins in the treatment of a mouse model of Niemann-Pick C disease

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Critical role for glycosphingolipids in Niemann-Pick disease type C.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4048128

Orphan products: an emerging trend in drug approvals

28 November 2018

1 reference

12 December 2020

Spending on new drug development1

1 reference

12 December 2020

How to revive breakthrough innovation in the pharmaceutical industry

1 reference

12 December 2020

Open innovation: the new face of pharmaceutical research and development

1 reference

12 December 2020

10.2174/1568026613666131127160118

Identifiers

DOI

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24283970%20AND%20SRC:MED&resulttype=core&format=json

1 March 2020

1 reference

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24283970%20AND%20SRC:MED&resulttype=core&format=json

1 March 2020

PubMed publication ID

24283970

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24283970%20AND%20SRC:MED&resulttype=core&format=json