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Orphan Diseases

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

Papers overview

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Highly Cited
2013
Highly Cited
2013
At mucosal surfaces, the immune system should not initiate inflammatory immune responses to the plethora of antigens constantly… Expand
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Highly Cited
2012
Highly Cited
2012
Advances in genomics over the past 20 years have enhanced the precision and efficiency of breeding programs1 in many temperate… Expand
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Highly Cited
2012
Highly Cited
2012
Orphan drug incentives have stimulated research into diseases with significant unmet medical need. Although the targeting of… Expand
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Highly Cited
2011
Highly Cited
2011
BackgroundOrphan drugs are a growing issue of importance to European healthcare policy makers. The success of orphan drug… Expand
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Review
2010
Review
2010
Sustained advocacy efforts driven by patients' organizations to make rare diseases a health priority have led to regulatory and… Expand
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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… Expand
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Review
2004
Review
2004
Pulmonary arterial hypertension is a disease of the small pulmonary arteries characterized by vascular narrowing and increased… Expand
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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… Expand
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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… Expand
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Highly Cited
1994
Highly Cited
1994
The cognitive and social developmental status of a representative group of Romanian orphans between the ages of 23 and 50 months… Expand
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