2 projets européens trouvés
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TERMINÉ
Preclinical study of Recombinant human Anti-C5 for the Treatment of atypical HUS (PRATH)
Date du début: 1 mai 2010, Date de fin: 30 avr. 2012,
...stem in September 2008. This molecule has been already characterized by in vitro and in vivo experiments and a complete preclinical study is the next step for the development of this potential orphan drug useful for the treatment of aHUS. Preclinical studies of this EU designated orphan medicinal product has been design by ADIENNE S.r.l. and will be perform in collaboration with the University of ...
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Participants partenaires
TERMINÉ
Smart multifunctional GLA-nanoformulation for Fabry disease (Smart-4-Fabry)
Date du début: 1 janv. 2017, Date de fin: 31 déc. 2020,
Lysosomal storage disorders (LSD) diseases are a group of rare diseases that currently lack a definitive cure. LSD incidence is about 1:5,000 - 1:10,000, representing a serious global health problem. In the case of Fabry LSD Disease (FD), the deficiency in α-Galactosidase A (GLA) enzyme activity results in the cellular accumulation of neutral glycosphingolipids, leading to widespread vasculopathy ...
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