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Drugs, Orphan

Known as: Drug, Orphan, Orphan drugs, drugs orphan 
A compound or product that demonstrates promise for the diagnosis and/or treatment of rare diseases and conditions that affect fewer than 200,000… 
National Institutes of Health

Papers overview

Semantic Scholar uses AI to extract papers important to this topic.
2013
2013
Impact of biomarker usage on oncology drug development
What is known and Objective:  The increasing cost of drug research and development and the decreasing number of new drugs being… 
2012
2012
Orphan Drugs for Rare Diseases
Orphan drugs are intended for diseases with a very low prevalence, and many countries have implemented legislation to support… 
Review
2012
Review
2012
Microbial L-Asparaginase and its future prospects
L-asparaginase hydrolyses the free L-asparagine and depletes it in blood and selectively drives the leukemic cells to death as… 
2011
2011
Clinical pharmacology as a cornerstone of orphan drug development
A recent US Food and Drug Administration (FDA) advisory committee meeting highlighted the potential of clinical pharmacology to… 
Review
2010
Review
2010
Emerging drugs for lysosomal storage diseases
Importance of the field: Because orphan drug regulations encouraged development of drugs for rare disorders by granting marketing… 
Highly Cited
2004
Highly Cited
2004
GHB (γ-Hydroxybutyrate) Carrier-Mediated Transport across the Blood-Brain Barrier
γ-Hydroxybutyrate (sodium oxybate, GHB) is an approved therapeutic agent for cataplexy with narcolepsy. GHB is widely abused as… 
Review
2004
Review
2004
Using pharmacogenetics and pharmacogenomics in the treatment of psychiatric disorders: some ethical and economic considerations
Current pharmacotherapies for psychiatric disorders are generally incompletely effective. Many patients do not respond well or… 
2002
2002
Gleevec for the treatment of chronic myelogenous leukemia: US. Food and Drug Administration regulatory mechanisms, accelerated approval, and orphan drug status.
Gleevec (imatinib mesylate), a highly promising new drug for the treatment of chronic myelogenous leukemia in blast crisis, in… 
Review
2001
Review
2001
Yohimbine in erectile dysfunction: would an orphan drug ever be properly assessed?
Abstract The systemic use of adrenergic antagonists in the treatment of sexual dysfunction has originated more controversy than… 
Highly Cited
1981
Highly Cited
1981
Herculine Barbin. Being the recently discovered memoirs of a nineteenth-century hermaphrodite
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