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, A; Frydman-Marom, A; Sharpe, N; van Duzer, J; Campbell, K CM; Vanhoutte, F (2019)
(e-pub ahead of print). Doi: 10.1002/cpdd.647