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1. (WO2019046244) METHODS OF ENHANCING AND/OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE
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Pub. No.: WO/2019/046244 International Application No.: PCT/US2018/048257
Publication Date: 07.03.2019 International Filing Date: 28.08.2018
IPC:
A61K 31/445 (2006.01) ,A61P 9/04 (2006.01) ,A61P 43/00 (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
435
having six-membered rings with one nitrogen as the only ring hetero atom
44
Non-condensed pyridines; Hydrogenated derivatives thereof
445
Non-condensed piperidines, e.g. piperocaine
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
9
Drugs for disorders of the cardiovascular system
04
Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43
Drugs for specific purposes, not provided for in groups A61P1/-A61P41/108
Applicants:
AMICUS THERAPEUTICS, INC. [US/US]; 1 Cedar Brook Drive Cranbury, New Jersey 08512, US
Inventors:
CASTELLI, Jeff; US
BARTH, Jay; US
SKUBAN, Nina; US
Agent:
ALEGRIA, Rory P.; US
Priority Data:
62/550,98428.08.2017US
Title (EN) METHODS OF ENHANCING AND/OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE
(FR) PROCÉDÉS POUR AMÉLIORER ET/OU STABILISER LA FONCTION CARDIAQUE CHEZ DES PATIENTS ATTEINTS DE LA MALADIE DE FABRY
Abstract:
(EN) Provided are methods for the treatment of Fabry disease in a patient. Certain methods relate to the treatment of ERT-experienced or ERT-naïve Fabry patients. Certain methods comprise administering to the patient about 100 mg to about 150 mg free base equivalent of migalastat for enhancing and/or stabilizing cardiac function.
(FR) L'invention concerne des procédés pour traiter la maladie de Fabry chez un patient. Certains procédés concernent le traitement de patients atteints de la maladie de Fabry soumis à une TES ou non soumis à une TES. Certains procédés consistent à administrer au patient environ 100 mg à environ 150 mg d'équivalent de base libre de migalastat pour améliorer et/ou stabiliser la fonction cardiaque.
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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, 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)