Ibiglustat (L-Malic acid) is a potent inhibitor of glucosylceramide synthase (GCS), demonstrating oral bioavailability and the ability to penetrate the blood-brain barrier. This compound is instrumental for research into various lysosomal storage disorders, including Gaucher disease type 3, Parkinson's disease linked to GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease. Its selective inhibition of GCS makes it a valuable tool for studying the biochemical pathways associated with these conditions.
Ibiglustat (L-Malic acid) is a potent inhibitor of glucosylceramide synthase (GCS), demonstrating oral bioavailability and the ability to penetrate the blood-brain barrier. This compound is instrumental for research into various lysosomal storage disorders, including Gaucher disease type 3, Parkinson's disease linked to GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease. Its selective inhibition of GCS makes it a valuable tool for studying the biochemical pathways associated with these conditions.
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