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1. WO2007150064 - METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF β-GLUCOCEREBROSIDASE

Publication Number WO/2007/150064
Publication Date 27.12.2007
International Application No. PCT/US2007/072016
International Filing Date 25.06.2007
IPC
A01N 43/40 2006.01
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
NPRESERVATION OF BODIES OF HUMANS OR ANIMALS OR PLANTS OR PARTS THEREOF; BIOCIDES, e.g. AS DISINFECTANTS, AS PESTICIDES OR AS HERBICIDES; PEST REPELLANTS OR ATTRACTANTS; PLANT GROWTH REGULATORS
43Biocides, pest repellants or attractants, or plant growth regulators containing heterocyclic compounds
34having rings with one nitrogen atom as the only ring hetero atom
40six-membered rings
A61K 31/445 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
44Non-condensed pyridines; Hydrogenated derivatives thereof
445Non-condensed piperidines, e.g. piperocaine
CPC
A61K 2300/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
2300Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
A61K 31/194
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
185Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic, hydroximic acids
19Carboxylic acids, e.g. valproic acid
194having two or more carboxyl groups, e.g. succinic, maleic or phthalic acid
A61K 31/437
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
4353ortho- or peri-condensed with heterocyclic ring systems
437the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
A61K 31/445
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
44Non condensed pyridines; Hydrogenated derivatives thereof
445Non condensed piperidines, e.g. piperocaine
A61K 31/45
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
44Non condensed pyridines; Hydrogenated derivatives thereof
445Non condensed piperidines, e.g. piperocaine
45having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
A61K 31/713
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
713Double-stranded nucleic acids or oligonucleotides
Applicants
  • AMICUS THERAPEUTICS, INC. [US]/[US] (AllExceptUS)
  • WUSTMAN, Brandon [US]/[US] (UsOnly)
Inventors
  • WUSTMAN, Brandon
Agents
  • FEHLNER, Paul, F.
Priority Data
60/815,95223.06.2006US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF β-GLUCOCEREBROSIDASE
(FR) PROCÉDÉ DESTINÉ AU TRAITEMENT DE TROUBLES NEUROLOGIQUES PAR INTENSIFICATION DE L'ACTIVITÉ DE LA β-GLUCOCÉRÉBROSIDASE
Abstract
(EN)
Provided is a method of increasing the stability of wild-type β- glucocerebrosidase. Also provided are methods of treating and/or preventing an individual having a neurological disease in which increased expression or activity of β-glucocerebrosidase in the central nervous system would be beneficial. This method comprises administering an effective amount of a pharmacologic chaperone for β- glucocerebrosidase, with the proviso that the individual does not have a mutation in the gene encoding β-glucocerebrosidase. Further provided are β-glucocerebrosidase inhibitors which have been identified as specific pharmacologic chaperones and which have been shown to increase activity of β-glucocerebrosidase in vivo in the central nervous system.
(FR)
La présente invention concerne un procédé permettant d'augmenter la stabilité du type sauvage de la β- glucocérébrosidase. L'invention concerne également des procédés de traitement et/ou de prévention d'une maladie neurologique chez un individu, pour lequel l'expression ou l'activité accrue de la β-glucocérébrosidase dans le système nerveux central devrait être bénéfique. Ce procédé consiste à administrer une quantité efficace d'un chaperon pharmacologique de la β-glucocérébrosidase, à la condition que l'individu ne présente pas de mutation au niveau du gène codant pour la β-glucocérébrosidase. L'invention concerne en outre des inhibiteurs de la β-glucocérébrosidase qui ont été identifiés comme chaperons pharmacologiques spécifiques et dont il a été démontré qu'ils intensifiaient l'activité de la β-glucocérébrosidase in vivo dans le système nerveux central.
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