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Furthermore, the GCLC floxed X CaMKII-Cre displays neurodegeneration without overexpressing pathology related genes

The Phase 3 ABTECT program comprised two double-blind, placebo-controlled induction RCTs (NCT05507203 and NCT05507216) testing 25mg and 50mg once-daily doses versus placebo in 1,275 patients across 600+ sites in 36 countries, with the primary endpoint of clinical remission at week 8, enrolling patients with inadequate response to conventional or advanced therapies including the largest JAK inhibitor-experienced population in UC trials (79)

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Among the diverse forms of genetic cardiomyopathy, ATTR-CM, hypertrophic cardiomyopathy (HCM), and arrhythmogenic right ventricular cardiomyopathy (ARVC) have garnered significant attention because of their high prevalence, well-defined genetic basis, and feasibility of gene-targeted therapies