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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
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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
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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.
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