Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX‐02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers - Leubitz, Andi; Frydman‐Marom, Anat; Sharpe, Neal; Duzer, John; Campbell, Kathleen C.M.; Vanhoutte, Frédéric (Clinical pharmacology in drug development, 2019)
Keywords: aminoglycoside; ELX‐02; genetic disorders; healthy volunteers; Mutation; nonsense mutations; Pharmacokinetics; phase 1; safety; single ascending dose; translational readthrough; single ascending dose; aminoglycoside; phase 1; translational readthrough; genetic disorders; safety; healthy volunteers; ELX-02; pharmacokinetics; nonsense mutations