elx02 sulfate

ELX-02 sulfate, also known as NB-124 sulfate, is a novel compound currently under development as a therapeutic agent for genetic diseases triggered by nonsense mutations. This investigational compound, an advanced synthetic eukaryotic ribosome selective glycoside (ERSG), shows promising potential for targeted treatment.

ELX-02, also known as NB-124, is a eukaryotic ribosomal selective glycoside (ERSG) designed to increase the read-through activity in patients with nonsense mutations and enable the production of sufficient amounts of full-length functional protein to rest

ELX-02 disulfate是一种合成的真核核糖体特异性糖苷 (ERSG)，能够诱导无义突变的翻译读透(read through)，产生正常定位的全长功能蛋白，可用于研究无义突变引起的遗传疾病。