[EN] PYRAZOLOPYRIDAZINES AND METHODS FOR TREATING RETINAL-DEGENERATIVE DISEASES AND HEARING LOSS ASSOCIATED WITH USHER SYNDROME<br/>[FR] PYRAZOLOPYRIDAZINES ET MÉTHODES DE TRAITEMENT DE MALADIES DÉGÉNÉRATIVES DE LA RÉTINE ET DE LA PERTE D'AUDITION ASSOCIÉE AU SYNDROME D'USHER
申请人:USHER III INITIATIVE INC
公开号:WO2014066836A1
公开(公告)日:2014-05-01
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.