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Creating the costliest orphan. The Orphan Drug Act in the development of Ceredase
scientific article published on 01 January 1992
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instance of
scholarly article
1 reference
stated in
Europe PubMed Central
PubMed publication ID
1464480
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:1464480%20AND%20SRC:MED&resulttype=core&format=json
retrieved
15 September 2019
title
Creating the costliest orphan. The Orphan Drug Act in the development of Ceredase
(English)
1 reference
stated in
Europe PubMed Central
PubMed publication ID
1464480
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:1464480%20AND%20SRC:MED&resulttype=core&format=json
retrieved
15 September 2019
main subject
orphan drug
1 reference
based on heuristic
inferred from title
author name string
D. P. Goldman
series ordinal
1
1 reference
stated in
Europe PubMed Central
PubMed publication ID
1464480
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A1464480
retrieved
29 September 2022
A. E. Clarke
series ordinal
2
1 reference
stated in
Europe PubMed Central
PubMed publication ID
1464480
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A1464480
retrieved
29 September 2022
A. M. Garber
series ordinal
3
1 reference
stated in
Europe PubMed Central
PubMed publication ID
1464480
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A1464480
retrieved
29 September 2022
language of work or name
English
1 reference
stated in
National Center for Biotechnology Information
PubMed publication ID
1464480
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/esummary.fcgi?db=pubmed&retmode=json&id=1464480
retrieved
29 September 2022
publication date
1 January 1992
1 reference
stated in
Europe PubMed Central
PubMed publication ID
1464480
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:1464480%20AND%20SRC:MED&resulttype=core&format=json
retrieved
15 September 2019
published in
International Journal of Technology Assessment in Health Care
1 reference
stated in
Europe PubMed Central
PubMed publication ID
1464480
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:1464480%20AND%20SRC:MED&resulttype=core&format=json
retrieved
15 September 2019
volume
8
1 reference
stated in
Europe PubMed Central
PubMed publication ID
1464480
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:1464480%20AND%20SRC:MED&resulttype=core&format=json
retrieved
15 September 2019
issue
4
1 reference
stated in
Europe PubMed Central
PubMed publication ID
1464480
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:1464480%20AND%20SRC:MED&resulttype=core&format=json
retrieved
15 September 2019
page(s)
583-597
1 reference
stated in
Europe PubMed Central
PubMed publication ID
1464480
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:1464480%20AND%20SRC:MED&resulttype=core&format=json
retrieved
15 September 2019
cites work
Purification of beta-glucocerebrosidase by preparative-scale high-performance liquid chromatography: the use of ethylene glycol-containing buffers for chromatography of hydrophobic glycoprotein enzymes
1 reference
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Crossref
reference URL
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7 January 2021
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Frequency of carriers of chronic (type I) Gaucher disease in Ashkenazi Jews
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Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
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7 January 2021
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Heterogeneity of mutations in the acid beta-glucosidase gene of Gaucher disease patients
1 reference
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Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
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7 January 2021
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Human lysosomal beta-glucosidase: purification by affinity chromatography
1 reference
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Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
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7 January 2021
based on heuristic
inferred from DOI database lookup
Uptake and distribution of placental glucocerebrosidase in rat hepatic cells and effects of sequential deglycosylation
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Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease
1 reference
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Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Enzyme replacement therapy in Gaucher's disease: large-scale purification of glucocerebrosidase suitable for human administration
1 reference
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Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Enzyme replacement therapy in Gaucher's disease: a rapid, high-yield method for purification of glucocerebrosidase
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Enzyme replacement therapy in Gaucher's disease: preliminary clinical trial of a new enzyme preparation
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Gaucher's Disease
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Purification of human placental glucocerebrosidase using a two-step high-performance hydrophobic and gel permeation column chromatography method
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Gaucher disease
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Beneficial effect of pre-transplant splenectomy on displacement bone marrow transplantation for Gaucher's syndrome
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
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7 January 2021
based on heuristic
inferred from DOI database lookup
Prevalent and rare mutations among Gaucher patients
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Gaucher's disease: lack of antibody response in 12 patients following repeated intravenous infusions of mannose terminal glucocerebrosidase.
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Replacement therapy for inherited enzyme deficiency. Use of purified glucocerebrosidase in Gaucher's disease.
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Treatment of Gaucher's disease with liposome-entrapped glucocerebroside: beta-glucosidase
1 reference
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Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
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7 January 2021
based on heuristic
inferred from DOI database lookup
Some of the tough decisions required by a national health plan
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Genetic heterogeneity in type 1 Gaucher disease: multiple genotypes in Ashkenazic and non-Ashkenazic individuals
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
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7 January 2021
based on heuristic
inferred from DOI database lookup
Evidence for receptor-mediated binding of glycoproteins, glycoconjugates, and lysosomal glycosidases by alveolar macrophages
1 reference
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Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
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7 January 2021
based on heuristic
inferred from DOI database lookup
Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
A mutation in the human glucocerebrosidase gene in neuronopathic Gaucher's disease
1 reference
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Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Splenic transplantation in a case of Gaucher's disease
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Regression of the radiological changes of Gaucher's disease following bone marrow transplantation
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Purification of glucosylceramidase by affinity chromatography.
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Prediction of severity of Gaucher's disease by identification of mutations at DNA level
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
A procedure for the rapid purification in high yield of human glucocerebrosidase using immunoaffinity chromatography with monoclonal antibodies
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Clinical and biochemical outcome of marrow transplantation for Gaucher disease of the Norrbottnian type
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1017%2FS0266462300002294
retrieved
7 January 2021
based on heuristic
inferred from DOI database lookup
Identifiers
DOI
10.1017/S0266462300002294
1 reference
stated in
Europe PubMed Central
PubMed publication ID
1464480
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:1464480%20AND%20SRC:MED&resulttype=core&format=json
retrieved
15 September 2019
PubMed publication ID
1464480
1 reference
stated in
Europe PubMed Central
PubMed publication ID
1464480
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:1464480%20AND%20SRC:MED&resulttype=core&format=json
retrieved
15 September 2019
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