Background and aims: Orphan diseases, or rare diseases, are defined in Europe as
diseases that affect less than 5 out of every 10,000 citizens. Given the small number of cases …

There are approximately 6000 rare diseases, and 80% of them are genetic. In Europe, a
disease is considered rare when it affects no more than one person in 2000. In France, two …

Not only in Europe and USA, but also in many other countries rare disorders—so-called
orphan diseases—have attracted more and more attention. The formation of specialized …

Abstract Around 3.9% of world population suffers with rare diseases, with roughly 25–30
million (almost 1 in 10) affected in the United States alone (National Institutes of Health …

The implementation of the European Union (EU) Regulation for orphan medicines has
resulted in> 1000 designations and> 70 marketing authorisations. Of those, in 21 cases …

The management of orphan rare diseases has been the goal of two successive government
plans since 2004. They allowed the management of these diseases to be handled initially …

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Though rare diseases affect fewer patients than common diseases, developing drugs for
them is subject to the same principles. But constructing and leading rare diseases clinical …

There are two major problems with the development of therapies for rare diseases. First,
among over 7000 such diseases, the vast majority are caused by genetic defects and/or …

Rare diseases are serious, chronically and/or progressively disabling, and can be life-
limiting and life-threatening. According to the US National Institutes of Health (NIH), there …