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Journal article

Drugs for rare disorders

Abstract:

Estimates of the frequencies of rare disorders vary from country to country; the global average defined prevalence is 40 per 100 000 (0.04%). Some occur in only one or a few patients. However, collectively rare disorders are fairly common, affecting 6-8% of the US population, or about 30 million people, and a similar number in the European Union. Most of them affect children and most are genetically determined. Diagnosis can be difficult, partly because of variable presentations and partly be...

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Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1111/bcp.13331

Authors


More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Primary Care Health Sciences
Oxford college:
Green Templeton College
Role:
Author
ORCID:
0000-0002-6800-5532
Publisher:
Wiley Publisher's website
Journal:
British Journal of Clinical Pharmacology Journal website
Volume:
83
Issue:
8
Pages:
1607-1613
Publication date:
2017-06-27
Acceptance date:
2017-05-10
DOI:
EISSN:
1365-2125
ISSN:
0306-5251
Pmid:
28653488
Source identifiers:
846621
Language:
English
Keywords:
Pubs id:
pubs:846621
UUID:
uuid:3b729bae-5d3c-481b-9b72-3891de451e61
Local pid:
pubs:846621
Deposit date:
2018-08-17

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