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,”
says Prof. Nicolas Lévy, head of the medical genetics department at the 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 it
“A priority health and research issue”
commensurate with their damage as well as the possibilities they open up, including with regard to the management of much more frequent pathologies.
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Duchenne muscular dystrophy: immense progress in treatment
The doctor also pleads for his great project: Giptis
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