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The unfolded protein response in amyotrophic later sclerosis: results of a phase 2 trial

23 Apr 2021

Abstract excerpt

Strong evidence suggests that endoplasmic reticulum stress plays a critical role in the pathogenesis of amyotrophic lateral sclerosis (ALS) through altered regulation of proteostasis. Robust preclinical findings demonstrated that guanabenz selectively inhibits endoplasmic reticulum stress-induced eIF2α-phosphatase, allowing misfolded protein clearance, reduces neuronal death and prolongs survival in in vitro and...

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PMID: 33905493
DOI: 10.1093/brain/awab167

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