Make rare diseases a priority issue

In Rare diseases and the hopes of the medicine of the future, the Pr Nicolas Lévy pleads for “A priority health and research issue”, equal to their damage and to the possibilities they open up.

Duchenne muscular dystrophy, progeria, cystic fibrosis or sarcoidosis … You have no doubt heard of it, and you may have said: “It only happens to others.” Rare diseases are however more frequent than one thinks: of course, each of them affects only a small number of patients; but, taken together, they affect 3 million people in France. That is 1 in 20 people suffering from a pathology that is often serious, rarely curable. “It’s considerable”, throw the Pr Nicolas Lévy, head of the medical genetics department at La Timone hospital (Marseille) and Inserm research director. With the journalist and documentary filmmaker Roland Portiche, he pleads, in Rare diseases and the hopes of the medicine of the future, to make “A priority health and research issue” up to their damage as well as the possibilities that they open up, including with regard to the management of much more frequent pathologies.

The doctor also pleads for his great project: Giptis

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