– life-threatening or chronically debilitating diseases which are of such low prevalence that special combined efforts are needed to address them –
that as many as 5000 to 7000 distinct rare diseases exist, affecting for most of them a very small number of patients.
A DISEASE OR DISORDER
is defined as rare in Europe when it affects fewer than 1 in 2000 people.
for most of these diseases, for most of these patients and their families, are still to be invented, registered and marketed.
RARE DISEASES ENVIRONMENT
ITALIAN ENVIRONMENT FOR RARE DISEASES