Lysosomal Storage Disorders Articles & Analysis
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Effectively reduced the production of inflammatory cytokines by GD macrophages. Gaucher Disease is a lysosomal storage disease characterized by deficient activity of the glucocerebrosidase enzyme (GCase) encoded by the GBA1 gene. ...
About Fabry Disease Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is necessary for metabolizing globotriaosylceramide (Gb3). ...
Capitalizing on significant advances in computational biology and drug discovery, the Company has applied its proprietary computational SEE-Tx® platform to generate its lead programs in GBA1 Parkinson’s disease, Gaucher disease as well as four additional programs to advance the treatment of lysosomal storage disorders. In addition, the ...
Feldman’s laboratory uses patient-derived induced pluripotent stem cells (iPSC) to model lysosomal storage diseases and for drug discovery. Samuel Broder, MD has spent a lifetime at the forefront of science and medicine in many diverse arenas. ...
About Fabry Disease Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is necessary for metabolizing globotriaosylceramide (Gb3). ...