Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX‐02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers
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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… read more here.
Keywords: caused nonsense; safety; increase; single ascending ... See more keywords