Description
Rare diseases affect hundreds of millions of people every year. Many of these are children: it is estimated that 1 in 5 is a child or adolescent. Often the consequences are serious and progressively debilitating, without specific treatment. About one third of the patients do not exceed the pediatric age.
Until a few years ago, in fact, there were no scientific or political activities aimed at research in the field of rare diseases. Today the scenario is changing: technological innovation on the one hand and biomedical research on the other have made available to the healthcare world and institutions opportunities for intervention capable of changing the natural history of many rare diseases.
Italy boasts advanced legislation on newborn screening: the panel currently has over 40 pathologies. But this is not enough. there is a need to raise awareness and generate change for millions of children around the world living with a rare disease, their families and their caregivers.
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