The best dart in the bullseye for the cure of rare diseases

Charo Barroso

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Moebius syndrome, Marfan syndrome, Brugada syndrome, X-Fragil syndrome, Hutchinson-Gilford progeria, Schönlein-Henoch purpura … in all probability, these names are unknown to most of the population, but they are well present in the life of those who suffer them. They are rare diseases, infrequent pathologies whose prevalence is below five in every ten thousand people, of which there are more than seven thousand in the world that affect seven percent of the population. In Europe we speak of thirty million people and, in Spain, of more than three million. Genetic diseases, many of them congenital, which usually appear from childhood, which lead to a high degree of chronic disability that undermines quality

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