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Rare or orphan diseases refer to a group of indications that are invariably life threatening or chronically debilitating and that presents with low prevalence (1∶1500-2500).In the USA and Europe,this prevalence translates to between 50 and 60 million affected individuals.Hence,although individually each of the diseases may be rare,as a group they can account for up to 10％ of the general population.More than 7000 different rare diseases have been described in the literature and of these,therapies have been approved for only approximately 5％,suggesting that there is a large measure of unmet medical need associated with this group of diseases.