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1. (WO2018201146) COMPOUNDS AND METHODS OF TREATING RETINAL DEGENERATION

Pub. No.:    WO/2018/201146    International Application No.:    PCT/US2018/030251
Publication Date: Fri Nov 02 00:59:59 CET 2018 International Filing Date: Tue May 01 01:59:59 CEST 2018
IPC: A61K 31/18
A61K 31/34
A61K 31/341
A61K 31/40
A61K 31/495
A61P 29/00
Applicants: CASE WESTERN RESERVE UNIVERSITY
UNIVERSITY OF PITTSBURGH
Inventors: PALCZEWSKI, Krzysztof
CHEN, Yuanyuan
LIPKOVICH, Holly
Title: COMPOUNDS AND METHODS OF TREATING RETINAL DEGENERATION
Abstract:
This application relates to compounds and methods of treating retinal degeneration associated with inherited rhodopsin mutations in the ocular tissue of a subject. The retinal degeneration, can include, for example, macular degeneration, including age-related macular degeneration, Stargardt disease, and retinitis pigmentosa. The retinitis pigmentosa can include autosomal dominate retinitis pigmentosa associated with a P23H RHO mutation. A method of treating retinal degeneration in a subject includes administering to the subject a therapeutically effective amount of a compound of formula (I), wherein the compound of formula (I) acts as a chaperone of rhodopsin.