Factors Contributing to the Efficacy-Effectiveness Gap in the Case of Orphan Drugs for Metabolic Diseases.

about

Factors Contributing to the Efficacy-Effectiveness Gap in the Case of Orphan Drugs for Metabolic Diseases.

Item

http://www.wikidata.org/entity/Q38656087

description

2017 nî lūn-bûn

@nan

2017年の論文

@ja

2017年学术文章

@wuu

2017年学术文章

@zh-cn

2017年学术文章

@zh-hans

2017年学术文章

@zh-my

2017年学术文章

@zh-sg

2017年學術文章

@yue

2017年學術文章

@zh

2017年學術文章

@zh-hant

name

Factors Contributing to the Ef ...... Drugs for Metabolic Diseases.

@en

type

Item

label

Factors Contributing to the Ef ...... Drugs for Metabolic Diseases.

@en

prefLabel

Factors Contributing to the Ef ...... Drugs for Metabolic Diseases.

@en

P1476

Factors Contributing to the Ef ...... Drugs for Metabolic Diseases.

@en

P2093

C C Gispen-de Wied

http://www.w3.org/2001/XMLSchema#string

C E M Hollak

http://www.w3.org/2001/XMLSchema#string

M Biegstraaten

http://www.w3.org/2001/XMLSchema#string

V Stoyanova-Beninska

http://www.w3.org/2001/XMLSchema#string

Y Schuller

http://www.w3.org/2001/XMLSchema#string

P2860

Effectiveness, safety and costs of orphan drugs: an evidence-based review

Biomarkers and surrogate endpoints: preferred definitions and conceptual framework

Development and validation of COMPASS: clinical evidence of orphan medicinal products - an assessment tool.

Rare diseases and the assessment of intervention: what sorts of clinical trials can we use?

European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene.

Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders.

Phenylalanine blood levels and clinical outcomes in phenylketonuria: a systematic literature review and meta-analysis.

Clinical evidence for orphan medicinal products-a cause for concern?

Clinical trials and rare diseases.

Determinants for successful marketing authorisation of orphan medicinal products in the EU.

P2888

s40265-017-0788-z

P31

scholarly article

P356

10.1007/S40265-017-0788-Z

http://www.w3.org/2001/XMLSchema#string

P577

2017-07-27T00:00:00Z

http://www.w3.org/2001/XMLSchema#dateTime

P6179

1090905314

http://www.w3.org/2001/XMLSchema#string

P698

28752290

http://www.w3.org/2001/XMLSchema#string

P921

orphan drug

P932

5577060

http://www.w3.org/2001/XMLSchema#string