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1. (WO2017040603) REGIMENS COMPRISING (3R, 4R, 5S)-5-(DIFLUOROMETHYL)PIPERIDINE-3.4-DIOL FOR TREATING AND PREVENTING LYSOSOMAL DISORDERS AND DEGENERATIVE DISORDERS OF THE CENTRAL NERVOUS SYSTEM
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Pub. No.: WO/2017/040603 International Application No.: PCT/US2016/049614
Publication Date: 09.03.2017 International Filing Date: 31.08.2016
IPC:
A61K 31/445 (2006.01) ,A61P 25/00 (2006.01) ,A61P 25/16 (2006.01) ,A61P 25/28 (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
25
Drugs for disorders of the nervous system
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
14
for treating abnormal movements, e.g. chorea, dyskinesia
16
Anti-Parkinson drugs
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
28
for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
Applicants:
AMICUS THERAPEUTICS, INC. [US/US]; 1 Cedar Brook Drive Cranbury, New Jersey 08512, US
Inventors:
CLARK, Sean; US
Agent:
ALEGRIA, Rory P.; US
Priority Data:
15/251,55930.08.2016US
62/212,29131.08.2015US
Title (EN) REGIMENS COMPRISING (3R, 4R, 5S)-5-(DIFLUOROMETHYL)PIPERIDINE-3.4-DIOL FOR TREATING AND PREVENTING LYSOSOMAL DISORDERS AND DEGENERATIVE DISORDERS OF THE CENTRAL NERVOUS SYSTEM
(FR) SCHÉMAS POSOLOGIQUES COMPRENANT (3R, 4R, 5S)-5-(DIFLUOROMÉTHYL)PIPÉRIDINE-3,4-DIOL POUR LE TRAITEMENT ET LA PRÉVENTION DE TROUBLES LYSOSOMIAUX ET DE TROUBLES DÉGÉNÉRATIFS DU SYSTÈME NERVEUX CENTRAL
Abstract:
(EN) Described herein are dosing regimens and kits comprising (3R, 4R, 5S)-5-(difluoromethyl)piperidine-3.4-diol for the treatment and/or prevention of lysosomal storage disorders such as Gaucher disease. Also described are dosing regimens and kits comprising (3R, 4R, 5S)-5-(difluoromethyl)piperidine-3.4-diol for the treatment and/or prevention of degenerative disorders of the central nervous system, such as the synucleinopathies Parkinson's disease or Lewy Body Dementia.
(FR) L'invention concerne des schémas et trousses posologiques comprenant (3R, 4R, 5S)-5-(difluorométhyl)pipéridine-3,4-diol pour le traitement et/ou la prévention de troubles de stockage lysosomial, comme la maladie de Gaucher. L'invention concerne également des schémas et trousses posologiques comprenant (3R, 4R, 5S)-5-(difluorométhyl)pipéridine-3,4-diol pour le traitement et/ou la prévention de troubles dégénératifs du système nerveux central, tels que les synucléinopathies comme la maladie de Parkinson ou la démence à corps de Lewy.
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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, 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 (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)