申请人:Eloxx Pharmaceuticals Ltd.
公开号:US20190016746A1
公开(公告)日:2019-01-17
Novel aminoglycosides, represented by Formulae I, Ia, III and IIIa, as defined in the instant specification, designed to exhibit stop codon mutation readthrough activity, are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic diseases and disorders, such as diseases and disorders associated with stop codon mutations.