(Q53202992)

English

Enzyme replacement therapy in orphan and ultra-orphan diseases: the limitations of standard economic metrics as exemplified by Fabry-Anderson disease.

scientific article published in January 2007

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scholarly article

1 reference

Europe PubMed Central

PubMed publication ID

12 May 2018

http://europepmc.org/abstract/MED/17335306

Enzyme replacement therapy in orphan and ultra-orphan diseases: the limitations of standard economic metrics as exemplified by Fabry-Anderson disease. (English)

1 reference

Europe PubMed Central

PubMed publication ID

12 May 2018

http://europepmc.org/abstract/MED/17335306

enzyme replacement therapy

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1 reference

based on heuristic

inferred from title

2

1 reference

Europe PubMed Central

PubMed publication ID

12 May 2018

http://europepmc.org/abstract/MED/17335306

Evelyn L. Forget

3

object named as

Evelyn L Forget

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Raphael Schiffmann

4

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author name string

David F Moore

1

1 reference

Europe PubMed Central

PubMed publication ID

12 May 2018

http://europepmc.org/abstract/MED/17335306

publication date

1 January 2007

1 reference

Europe PubMed Central

PubMed publication ID

12 May 2018

http://europepmc.org/abstract/MED/17335306

PharmacoEconomics

1 reference

Europe PubMed Central

PubMed publication ID

12 May 2018

http://europepmc.org/abstract/MED/17335306

25

1 reference

Europe PubMed Central

PubMed publication ID

12 May 2018

http://europepmc.org/abstract/MED/17335306

3

1 reference

Europe PubMed Central

PubMed publication ID

12 May 2018

http://europepmc.org/abstract/MED/17335306

201-208

1 reference

Europe PubMed Central

PubMed publication ID

12 May 2018

http://europepmc.org/abstract/MED/17335306

https://scigraph.springernature.com/pub.10.2165/00019053-200725030-00003

0 references

Ein Beitrag zur Kenntniss der Purpura haemorrhagica nodularis (Purpura papulosa haemorrhagica Hebrae)

1 reference

https://api.crossref.org/works/10.2165%2F00019053-200725030-00003

21 January 2018

Drugs for exceptionally rare diseases: do they deserve special status for funding?

1 reference

https://api.crossref.org/works/10.2165%2F00019053-200725030-00003

21 January 2018

Methods in health service research. An introduction to bayesian methods in health technology assessment

1 reference

https://api.crossref.org/works/10.2165%2F00019053-200725030-00003

21 January 2018

A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1.

1 reference

https://api.crossref.org/works/10.2165%2F00019053-200725030-00003

21 January 2018

Fabry disease: overall effects of agalsidase alfa treatment

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Enzymatic Defect in Fabry's Disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Ethics, policy, and rare genetic disorders: the case of Gaucher disease in Israel

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Using and interpreting cost-effectiveness acceptability curves: an example using data from a trial of management strategies for atrial fibrillation

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

The Mainz Severity Score Index: a new instrument for quantifying the Anderson-Fabry disease phenotype, and the response of patients to enzyme replacement therapy

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Limits to health care: fair procedures, democratic deliberation, and the legitimacy problem for insurers.

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

A Bayesian approach to stochastic cost-effectiveness analysis. An illustration and application to blood pressure control in type 2 diabetes.

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Storage solutions: treating lysosomal disorders of the brain

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Willingness to pay for a quality-adjusted life year: implications for societal health care resource allocation

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Problems with UK government's risk sharing scheme for assessing drugs for multiple sclerosis

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Using the net benefit regression framework to construct cost-effectiveness acceptability curves: an example using data from a trial of external loop recorders versus Holter monitoring for ambulatory monitoring of "community acquired" syncope

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Prevalence of lysosomal storage disorders

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Assessment of health-related quality-of-life in males with Anderson Fabry Disease before therapeutic intervention

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Development of Orphan Vaccines: An Industry Perspective

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Modelling valuations for Eq-5d health states: an alternative model using differences in valuations.

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Orphan products: origins, progress, and prospects

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Time preference, duration and health state valuations

1 reference

https://pubmed.ncbi.nlm.nih.gov/17335306

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.2165/00019053-200725030-00003

1 reference

Europe PubMed Central

PubMed publication ID

12 May 2018

http://europepmc.org/abstract/MED/17335306

Dimensions Publication ID

0 references

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

12 May 2018

http://europepmc.org/abstract/MED/17335306

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