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1. (WO2013148103) NOVEL COMPOUNDS FOR PREVENTING AND/OR TREATING LYSOSOMAL STORAGE DISORDERS AND/OR DEGENERATIVE DISORDERS OF THE CENTRAL NERVOUS SYSTEM
Latest bibliographic data on file with the International Bureau   

Pub. No.: WO/2013/148103 International Application No.: PCT/US2013/029612
Publication Date: 03.10.2013 International Filing Date: 07.03.2013
IPC:
C07D 211/44 (2006.01) ,A61K 31/445 (2006.01) ,A61P 25/00 (2006.01)
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
211
Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
04
with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
06
having no double bonds between ring members or between ring members and non-ring members
36
with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
40
Oxygen atoms
44
attached in position 4
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
25
Drugs for disorders of the nervous system
Applicants: AMICUS THERAPEUTICS, INC.[US/US]; 1 Cedarbrook Drive Cranbury, New Jersey 08512, US
Inventors: BOYD, Robert; US
RYBCZYNSKI, Philip, J.; US
SHETH, Kamlesh; US
Agent: ALEGRIA, Rory, P.; Servilla Whitney LLC 33 Wood Avenue South Second Floor, Suite 210 Iselin, New Jersey 08830, US
Priority Data:
61/616,15927.03.2012US
Title (EN) NOVEL COMPOUNDS FOR PREVENTING AND/OR TREATING LYSOSOMAL STORAGE DISORDERS AND/OR DEGENERATIVE DISORDERS OF THE CENTRAL NERVOUS SYSTEM
(FR) NOUVEAUX COMPOSÉS POUR LA PRÉVENTION ET/OU LE TRAITEMENT DES TROUBLES DE STOCKAGE DES LYSOSOMES ET/OU DES TROUBLES DÉGÉNÉRATIFS DU SYSTÈME NERVEUX CENTRAL
Abstract:
(EN) Described are novel salts of the compound (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol, as well as methods of using the same for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease and/or Parkinson's disease.
(FR) L'invention concerne de nouveaux sels du composé (3R,4R,5S)-5- (difluorométhyl)pipéridine-3,4-diol, ainsi que des méthodes d'utilisation de ceux-ci pour la prévention et/ou le traitement des troubles de stockage des lysosomes et/ou des troubles dégénératifs du système nerveux central. En particulier, la présente invention concerne des méthodes de prévention et/ou traitement de la maladie de Gaucher et/ou de la maladie de Parkinson.
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 (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, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)