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Article

Effects of PB‐TURSO on the transcriptional and metabolic landscape of sporadic ALS fibroblasts

9 Sept 2022

Abstract excerpt

OBJECTIVE: ALS is a rapidly progressive, fatal disorder caused by motor neuron degeneration, for which there is a great unmet therapeutic need. AMX0035, a combination of sodium phenylbutyrate (PB) and taurursodiol (TUDCA, TURSO), has shown promising results in early ALS clinical trials, but its mechanisms of action remain to be elucidated. Therefore, our goal was to obtain an unbiased landscape of the molecular...

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PMID: 36083004
DOI: 10.1002/acn3.51648

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