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1. (WO2014066836) PYRAZOLOPYRIDAZINES AND METHODS FOR TREATING RETINAL-DEGENERATIVE DISEASES AND HEARING LOSS ASSOCIATED WITH USHER SYNDROME
Latest bibliographic data on file with the International Bureau   

Pub. No.: WO/2014/066836 International Application No.: PCT/US2013/066939
Publication Date: 01.05.2014 International Filing Date: 25.10.2013
IPC:
A61K 31/5025 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
33
Heterocyclic compounds
395
having nitrogen as a ring hetero atom, e.g. guanethidine, rifamycins
495
having six-membered rings with two nitrogen atoms as the only ring hetero atoms, e.g. piperazine
50
Pyridazines; Hydrogenated pyridazines
5025
ortho- or peri-condensed with heterocyclic ring systems
Applicants:
USHER III INITIATIVE, INC. [US/US]; 191 North Wacker Drive Suite 2090 Chicago, Illinois 60606, US
Inventors:
BURLI, Roland Werner; GB
ESMIEU, William Rameshchandra Krishna; GB
LOCK, Christopher James; GB
MALAGU, Karine Fabienne; GB
OWENS, Andrew Pate; GB
HARTE, William Edward; US
Agent:
LANGER, Matthew E.; Cooley LLP 1299 Pennsylvania Ave Suite 700 Washington, District of Columbia 20004, US
Priority Data:
61/718,61125.10.2012US
61/775,40208.03.2013US
Title (EN) PYRAZOLOPYRIDAZINES AND METHODS FOR TREATING RETINAL-DEGENERATIVE DISEASES AND HEARING LOSS ASSOCIATED WITH USHER SYNDROME
(FR) PYRAZOLOPYRIDAZINES ET MÉTHODES DE TRAITEMENT DE MALADIES DÉGÉNÉRATIVES DE LA RÉTINE ET DE LA PERTE D'AUDITION ASSOCIÉE AU SYNDROME D'USHER
Abstract:
(EN) 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.
(FR) La présente invention concerne des composés, des compositions et des méthodes pour le traitement de maladies dégénératives de la rétine, telles que la rétinite pigmentaire, l'amaurose congénitale de Leber, des dégénérescences rétiniennes syndromiques, la dégénérescence maculaire liée à l'âge et le syndrome d'Usher, ainsi que la perte d'audition associée au syndrome d'Usher. Le syndrome d'Usher qui est une maladie génétique et une cause majeure de surdité et de cécité, est associé à une mutation dans un gène quelconque parmi dix gènes.
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Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, KZ, LA, LC, LK, LR, LS, LT, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)