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The research base draws on Phase 2/3 trials, mechanistic studies, and metabolic outcomes research across multiple institutions
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(Luciana Chessa, Italy) Le professeur Luciana Chessa, partir des patients prsentant une forme particulire de la maladie (certaines personnes tant diagnostiqus alors qu'elles sont adultes), propose d'essayer de faire une classification des malades en fonction de leur mutation gntique et de leurs symptmes (relation gnotype phnotype)
