(Q33684105)

28 July 2017

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease (English)

1 reference

enzyme replacement therapy

28 July 2017

0 references

1 reference

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24884717%20AND%20SRC:MED&resulttype=core&format=json

6 June 2020

author name string

Iris Hoogenboom-Plug

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24884717%20AND%20SRC:MED&resulttype=core&format=json

6 June 2020

Maureen P M H Rutten-Van Mölken

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24884717%20AND%20SRC:MED&resulttype=core&format=json

6 June 2020

W Ken Redekop

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24884717%20AND%20SRC:MED&resulttype=core&format=json

6 June 2020

Ans T van der Ploeg

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24884717%20AND%20SRC:MED&resulttype=core&format=json

6 June 2020

Leona Hakkaart

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24884717%20AND%20SRC:MED&resulttype=core&format=json

6 June 2020

publication date

16 May 2014

1 reference

Orphanet Journal of Rare Diseases

28 July 2017

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24884717%20AND%20SRC:MED&resulttype=core&format=json

6 June 2020

volume

9

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24884717%20AND%20SRC:MED&resulttype=core&format=json

6 June 2020

page(s)

75

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24884717%20AND%20SRC:MED&resulttype=core&format=json

https://scigraph.springernature.com/pub.10.1186/1750-1172-9-75

6 June 2020

exact match

0 references

cites work

Survival analysis for economic evaluations alongside clinical trials--extrapolation with patient-level data: inconsistencies, limitations, and a practical guide

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

Estimating the budget impact of orphan medicines in Europe: 2010 - 2020.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

Burden of illness of Pompe disease in patients only receiving supportive care.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

Review of regulatory recommendations for orphan drug submissions in the Netherlands and Scotland: focus on the underlying pharmacoeconomic evaluations

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

Societal views on orphan drugs: cross sectional survey of Norwegians aged 40 to 67

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

Orphan drugs and the NHS: should we value rarity?

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

Pompe disease in infants and children

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

Frequency of glycogen storage disease type II in The Netherlands: implications for diagnosis and genetic counselling

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

EuroQol--a new facility for the measurement of health-related quality of life

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

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

21 June 2018

1 reference

Crossref

https://api.crossref.org/works/10.1186%2F1750-1172-9-75

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.

21 January 2018

1 reference

Crossref

https://api.crossref.org/works/10.1186%2F1750-1172-9-75

Challenges in health state valuation in paediatric economic evaluation: are QALYs contraindicated?

21 January 2018

1 reference

Crossref

https://api.crossref.org/works/10.1186%2F1750-1172-9-75

Evidence and values: requirements for public reimbursement of drugs for rare diseases--a case study in oncology.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4038090

The Dutch tariff: results and arguments for an effective design for national EQ-5D valuation studies

30 October 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/24884717

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Assessing the economic challenges posed by orphan drugs

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/24884717

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Coverage with evidence development: an examination of conceptual and policy issues

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/24884717

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1186/1750-1172-9-75

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24884717%20AND%20SRC:MED&resulttype=core&format=json

Dimensions Publication ID

6 June 2020

0 references

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24884717%20AND%20SRC:MED&resulttype=core&format=json

6 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24884717%20AND%20SRC:MED&resulttype=core&format=json