Orphan Diseases

Known as: Diseases, Orphan, disease orphans, Orphan Disease 
Rare diseases that have not been well studied.
National Institutes of Health

Topic mentions per year

Topic mentions per year

1975-2018
05019752018

Papers overview

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Review
2016
Review
2016
Aging is a natural process characterized by the declining ability of the different organs and tissues to respond to stress… (More)
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Review
2015
Review
2015
INTRODUCTION Several orphan drugs have been approved by the European Medicines Agency (EMA) over the past two decades. However… (More)
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2012
2012
To assess the methodological quality of Orphan Medicinal Product (OMP) dossiers and discuss possible reasons for the small number… (More)
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Highly Cited
2011
Highly Cited
2011
Phenotypes are investigated in model organisms to understand and reveal the molecular mechanisms underlying disease. Phenotype… (More)
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2011
2011
The low prevalence rate of orphan diseases (OD) requires special combined efforts to improve diagnosis, prevention, and discovery… (More)
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Highly Cited
2006
Highly Cited
2006
RATIONALE Pulmonary arterial hypertension (PAH) is an orphan disease for which the trend is for management in designated centers… (More)
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Highly Cited
2004
Highly Cited
2004
Primary ciliary dyskinesia (PCD) is a genetic disease characterized by abnormalities in ciliary structure/function. We… (More)
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Highly Cited
2001
Highly Cited
2001
Early global deprivation of institutionalized children may result in persistent specific cognitive and behavioral deficits. In… (More)
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Highly Cited
1997
Highly Cited
1997
The prognosis and risk factors for bronchiectasis are at present poorly known. The aim of this study was to examine the long-term… (More)
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1994
1994
The cognitive and social developmental status of a representative group of Romanian orphans between the ages of 23 and 50 months… (More)
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