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Tideglusib is a small molecule of the thiadiazolidinone class that acts as a potent, irreversible, non-ATP competitive inhibitor of glycogen synthase kinase 3 beta (GSK-3β). It was developed primarily for neurodegenerative diseases such as Alzheimer's disease and progressive supranuclear palsy (PSP), based on its ability to inhibit GSK-3β—a kinase implicated in tau hyperphosphorylation and amyloid plaque formation. Preclinical studies demonstrated neuroprotective effects including reduced tau phosphorylation, decreased amyloid deposition, prevention of neuronal loss, and improved cognitive function in animal models. Tideglusib has also been investigated for other indications such as myotonic dystrophy type I, tooth repair via dentine regeneration, amyotrophic lateral sclerosis (ALS), arrhythmogenic right ventricular cardiomyopathy, neuroblastoma, and ovarian cancer. Clinical trials in Alzheimer's disease and PSP were discontinued due to lack of efficacy, but research continues for other potential applications.
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