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Published in 2019 at "Clinical Pharmacology in Drug Development"
DOI: 10.1002/cpdd.647
Abstract: ELX‐02 is an investigational synthetic eukaryotic ribosome–selective glycoside optimized as a translational read‐through molecule that induces read through of nonsense mutations, resulting in normally localized full‐length functional proteins. ELX‐02 is being developed as a therapy…
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Keywords:
caused nonsense;
safety;
increase;
single ascending ... See more keywords