ピラゾロピリダジンおよび網膜変性疾患およびアッシャー症候群に伴う聴覚損失を治療するための方法

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.