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1. (WO2013158616) TREATMENT OF MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
Latest bibliographic data on file with the International Bureau   

Pub. No.:    WO/2013/158616    International Application No.:    PCT/US2013/036739
Publication Date: 24.10.2013 International Filing Date: 16.04.2013
IPC:
A61K 31/06 (2006.01), A61K 31/12 (2006.01), A61P 3/00 (2006.01)
Applicants: UNIVERSITY OF PITTSBURGH - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION [US/US]; 200 Gardner Steel Conference Center Pittsburgh, PA 15260 (US)
Inventors: MOHSEN MOHAMED, Al-Walid; (US).
VOCKLEY, Gerard; (US)
Agent: KOLE, Lisa, B.; Baker Botts L.L.P. 30 Rockefeller Plaza New York, NY 10112-4498 (US)
Priority Data:
61/624,864 16.04.2012 US
Title (EN) TREATMENT OF MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
(FR) TRAITEMENT DU DÉFICIT EN ACYL-COA-DÉSHYDROGÉNASE DES ACIDES GRAS À CHAÎNE MOYENNE
Abstract: front page image
(EN)The present invention provides for methods and compositions for treating medium chain acyl-CoA dehydrogenase deficiency. It is based, at least in part, on the discovery that phenylbutyrate can serve as a substrate for medium chain acyl-CoA dehydrogenase. In non-limiting embodiments, phenylbutyrate and/or another source of phenylacetate is administered as a chaperone treatment to patients suffering from medium chain acyl-CoA dehydrogenase deficiency.
(FR)L'invention concerne des procédés et des compositions destinés au traitement du déficit en acyl-CoA-déshydrogénase des acides gras à chaîne moyenne. L'invention repose, en partie, sur la découverte que le phénylbutyrate peut servir de substrat pour l'acyl-CoA-déshydrogénase des acides gras à chaîne moyenne. Dans des modes de réalisations non limitatifs, du phénylbutyrate et/ou une autre source de phénylacétate sont administrés comme thérapie chaperon à des patients souffrant d'un déficit en acyl-CoA-déshydrogénase des acides gras à chaîne moyenne.
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, IS, JP, KE, KG, KM, 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, 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, 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, ML, MR, NE, SN, TD, TG).
Publication Language: English (EN)
Filing Language: English (EN)