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1. (WO2019006164) COMBINATION THERAPY TO TREAT UREA CYCLE DISORDERS
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Pub. No.: WO/2019/006164 International Application No.: PCT/US2018/040088
Publication Date: 03.01.2019 International Filing Date: 28.06.2018
IPC:
A61K 48/00 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Applicants:
BAYLOR COLLEGE OF MEDICINE [US/US]; One Baylor Plaza Houston, TX 77030, US
Inventors:
MARINI, Juan C.; US
NAGAMANI, Sandesh Chakravarthy Sreenath; US
Agent:
SISTRUNK, Melissa L.; US
SHISHIMA, Gina; US
SMITH, Darren; US
Priority Data:
62/526,05428.06.2017US
Title (EN) COMBINATION THERAPY TO TREAT UREA CYCLE DISORDERS
(FR) POLYTHÉRAPIE POUR TRAITER DES TROUBLES DU CYCLE DE L'URÉE
Abstract:
(EN) Embodiments of the disclosure include certain formulations for methods of treating urea cycle disorders. The methods encompass compositions that comprise benzoate and phenylbutyrate that may be at certain doses and have certain ratios of the components. The benzoate and phenylbutyrate may act synergistically in treatment of the urea cycle disorders, in particular embodiments.
(FR) Des modes de réalisation de l'invention comprennent certaines formulations pour des méthodes de traitement de troubles du cycle de l'urée. Les méthodes comprennent des compositions qui comprennent du benzoate et du phénylbutyrate qui peuvent être à certaines doses et présentent certains rapports des composants. Le benzoate et le phénylbutyrate peuvent agir de manière synergique dans le traitement des troubles du cycle de l'urée, dans des modes de réalisation particuliers.
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, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, 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 (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, 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)