منابع مشابه
Orphan kidney diseases.
Rare kidney diseases are a unique subset of renal disorders that are often termed 'orphan' as a result of a multitude of reasons: the small number of patients with the consequent lack of well-defined natural history and course of many of these diseases, limited awareness among the medical community, and finally the significant cost of developing novel therapeutics which makes many of these dise...
متن کاملRare diseases and orphan drugs.
According to the Regulation (EC) N. 141/2000 of the European Parliament and of the Council, rare diseases are life-threatening or chronically debilitating conditions, affecting no more than 5 in 10 000 persons in the European Community. It is estimated that between 6000 to 8000 distinct rare diseases affect up to 6% of the total EU population. Therefore, these conditions can be considered rare ...
متن کاملDrug repositioning for orphan diseases
The need and opportunity to discover therapeutics for rare or orphan diseases are enormous. Due to limited prevalence and/or commercial potential, of the approximately 6000 orphan diseases (defined by the FDA Orphan Drug Act as <200 000 US prevalence), only a small fraction (5%) is of interest to the biopharmaceutical industry. The fact that drug development is complicated, time-consuming and e...
متن کاملFinancing drug discovery for orphan diseases.
Recently proposed 'megafund' financing methods for funding translational medicine and drug development require billions of dollars in capital per megafund to de-risk the drug discovery process enough to issue long-term bonds. Here, we demonstrate that the same financing methods can be applied to orphan drug development but, because of the unique nature of orphan diseases and therapeutics (lower...
متن کاملGlobal effort against rare and orphan diseases.
T he fifth International Congress on Rare Pulmonary Diseases and Orphan Drugs will take place in Milan, Italy, on February 8–9, 2013 (www.pulmonaryrarediseases. com). 10 yrs have passed since the first Congress was held. Since then, the Congress has taken place regularly every 2 yrs, with growing success and ever increasing numbers of participants and speakers from around the world. The Interna...
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ژورنال
عنوان ژورنال: Nephron
سال: 2012
ISSN: 1660-8151,2235-3186
DOI: 10.1159/000339785