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The Phase Ib trial is set to test a gene therapy designed to target a rare disorder that causes muscle and respiratory failure.

Trial aims to establish safety and provide proof of concept for GYS1 inhibition as promising new treatment strategy in patients with late-onset Pompe disease ...

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By depleting PTG, an activator of the glycogen chain-elongating enzyme glycogen synthase (GYS1), in laforin- and malin-deficient LD mice, we show that abnormal glycogen chain lengths and not ...

micro-RNA-mediated inhibition of p27 translation emerges as a novel mechanism that can reduce p27 in some human cancers. p27 proteolysis is initiated by several different mechanisms. Tyrosine (Tyr ...

A separate retrospective analysis of a larger cohort of 1091 patients with advanced NSCLC who were progression-free after at least 2 years of immune checkpoint inhibitor therapy similarly did not find ...

Hence, pathways that regulate macrophage phenotype are of strategic interest, with reprogramming strategies focusing on inhibitors of phosphoinositide 3-kinase-gamma (PI3Kγ) due to restricted immune ...

Elraglusib is aimed at treating pancreatic ductal adenocarcinoma. Credit: Jo Panuwat D/Shutterstock. Actuate Therapeutics has gained orphan medicinal product designation (OMPD) from the European ...

Department of Pharmacology and Molecular Sciences, Johns Hopkins University School of Medicine, Baltimore, Maryland 21205, United States Chemistry-Biology Interface Graduate Training Program, Johns ...

Faculty of Chemistry and Earth Sciences, Institute for Organic Chemistry and Macromolecular Chemistry, Friedrich Schiller University Jena, 07743 Jena, Germany Department of Paleobiotechnology, Leibniz ...