首页> 外国专利> Pyrazolopyridazines and their use for treating retinal-degenerative diseases and hearing loss associated with usher syndrome

Pyrazolopyridazines and their use for treating retinal-degenerative diseases and hearing loss associated with usher syndrome

机译:吡唑并哒嗪及其在治疗视网膜退行性疾病和与迎来综合征相关的听力损失中的用途

摘要

Compounds, compositions and methods for the treatment of retinal degenerative diseases, such as retinitis pigmentosa, Leber's congenital Amaurosis, Syndromic retinal degenerations, age-related macular degeneration and Usher Syndrome, and hearing loss associated with Usher Syndrome are described herein. Usher Syndrome, a rare genetic disorder and a leading cause of deafness and blindness, is associated with a mutation in any one of ten genes.
机译:本文描述了用于治疗视网膜退行性疾病的化合物,组合物和方法,例如色素性视网膜炎,Leber先天性阿莫罗病,综合征性视网膜变性,年龄相关性黄斑变性和Usher综合征以及与Usher综合征相关的听力丧失。厄舍氏综合症是一种罕见的遗传疾病,是耳聋和失明的主要原因,它与十个基因中任何一个的突变有关。

著录项

  • 公开/公告号IL238288A

    专利类型

  • 公开/公告日2018-04-30

    原文格式PDF

  • 申请/专利权人 USHER III INITIATIVE INC.;

    申请/专利号IL20150238288

  • 发明设计人

    申请日2015-04-14

  • 分类号A61K;A61P;C07D;

  • 国家 IL

  • 入库时间 2022-08-21 12:52:27

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