Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX-02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers.
Clinical pharmacology in drug development（2019）
关键词
ELX-02,safety,pharmacokinetics,single ascending dose,phase 1,healthy volunteers,genetic disorders,nonsense mutations,aminoglycoside,translational readthrough
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