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Published in 2020 at "Expert Opinion on Investigational Drugs"
DOI: 10.1080/13543784.2020.1828862
Abstract: ABSTRACT Introduction ELX-02, an investigational compound that is structurally an aminoglycoside analog, induces read-through of nonsense mutations through interaction with the ribosome, through which full-length functional proteins can be produced. It is being developed as…
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Keywords:
treatment;
elx investigational;
disease;
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