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

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.

主权项

1. A method for treating a retinal degenerative disease, comprising administering to a subject in need thereof an effective amount of a compound having the structure:or a pharmaceutically acceptable salt thereof.