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Transgenic mice overexpressing SNCA A53T displayed progressively deteriorated motor coordination functions in rotarod and pole climbing tests (Figure 1, A and B) and additionally displayed arrhythmic walking patterns (Supplemental Figure 1, AI

Early small trials suggested that TUDCA might slow functional decline in ALS patients, leading to larger studies.38 TUDCA was later tested in combination with sodium phenylbutyrate (marketed as Relyvrio/AMX0035)

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Several mutations in the bilirubin UDP-glucuronosyltransferase ( UGT1 ) gene of CN1 and CN2 patients have been identified, which result in complete inactivation of this enzyme in CN1 patients and markedly reduced glucuronidation in CN2 patients

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