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Smart multifunctional GLA-nanoformulation for Fabry disease (Smart-4-Fabry)
Start date: Jan 1, 2017, End date: Dec 31, 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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