Invention Application
WO2017118968A1 AMINOGLYCOSIDE DERIVATIVES AND USES THEREOF IN TREATING GENETIC DISORDERS 审中-公开
氨基糖苷衍生物及其在治疗遗传性疾病中的用途

AMINOGLYCOSIDE DERIVATIVES AND USES THEREOF IN TREATING GENETIC DISORDERS
Abstract:
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.
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