irbmA disease is considered rare when it affects no more than 5 people amongst 10,000 inhabitants. The low incidence of these diseases amongst the population, however, does not signify that few rare diseases exist. This in fact is a phenomena that affects millions of people in Italy alone, and tens of thousands throughout Europe. The number of rare diseases known and diagnosed to date is between 7,000 and 8,000.

The market for drugs to cure each of these single diseases is so small that the pharmaceutical industry has so far been reluctant to invest in the research and discovery processes required to develop effective treatments. As a consequence, the majority of these diseases remain without treatments or a cure, opening up an enormous gap and the need for new therapies.

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