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Smart-4-Fabry focuses on developing a novel nanoformulation of the enzyme α-Galactosidase A for treating Fabry disease. This approach aims to enhance the delivery and efficacy of the enzyme, addressing significant challenges in current therapies for rare diseases.
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 with particular detriment to the kidneys, heart and nervous system.
The current treatment for FD is…
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