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1. (WO2016100385) COMPOUNDS, COMPOSITIONS AND METHODS FOR TREATING OR PREVENTING NEURODEGENERATIVE DISORDERS
Latest bibliographic data on file with the International Bureau   

Pub. No.:    WO/2016/100385    International Application No.:    PCT/US2015/065885
Publication Date: 23.06.2016 International Filing Date: 15.12.2015
IPC:
A61K 31/519 (2006.01), A61K 45/06 (2006.01), A61P 25/28 (2006.01)
Applicants: PRESIDENT AND FELLOWS OF HARVARD COLLEGE [US/US]; 17 Quincy Street Cambridge, MA 02138 (US)
Inventors: RUBIN, Lee, L.; (US)
Agent: WARREN, Lisa, M.; (US)
Priority Data:
14/571,005 15.12.2014 US
Title (EN) COMPOUNDS, COMPOSITIONS AND METHODS FOR TREATING OR PREVENTING NEURODEGENERATIVE DISORDERS
(FR) COMPOSÉS, COMPOSITIONS ET MÉTHODES DE TRAITEMENT DE TROUBLES NEURODÉGÉNÉRATIFS
Abstract: front page image
(EN)The disclosure provides compounds, compositions, and methods for promoting motor neuron survival, and for treating or preventing neurodegenerative disorders, such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). In accordance with an embodiment of the present invention, a method of regulating survival of motor neuron (SMN) protein levels in a cell is provided. The method includes contacting the cell with an effective amount of an agent which inhibits the level or activity of Nedd8 activating enzyme (NAE) in the cell and an effective amount of an agent which increases the level of SM7N protein, thereby regulating SMN protein levels in the cell.
(FR)La présente invention concerne des composés, des compositions et des procédés pour favoriser la survie des motoneurones, et pour traiter ou prévenir les troubles neurodégénératifs, tels que l'atrophie musculaire spinale (SMA) et la sclérose latérale amyotrophique (ALS).
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, 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 (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (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)