BURLI, ROLAND WERNER,ESMIEU, WILLIAM RAMESHCHANDRA KRISHNA,LOCK, CHRISTOPHER JAMES,MALAGU, KARINE FABIENNE,OWENS, ANDREW PATE,HARTE, WILLIAM EDWARD
申请号：
EP13848995
公开号：
EP2911671A4
申请日:
2013.10.25
申请国别(地区):
EP
年份:
2016
代理人:
摘要：
Compounds, compositions and methods for the treatment of retinal degenerative diseases, such as retinitis pigmentosa, Lebers 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.
