PT2040548E - Método para o tratamento de distúrbios neurológicos através do aumento da atividade da -glucocerebrosidase - Google Patents

Método para o tratamento de distúrbios neurológicos através do aumento da atividade da -glucocerebrosidase Download PDF

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Publication number
PT2040548E
PT2040548E PT07812295T PT07812295T PT2040548E PT 2040548 E PT2040548 E PT 2040548E PT 07812295 T PT07812295 T PT 07812295T PT 07812295 T PT07812295 T PT 07812295T PT 2040548 E PT2040548 E PT 2040548E
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quot
substituted
isofagomine
unsubstituted
gcase
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PT07812295T
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English (en)
Portuguese (pt)
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Brandon Wustman
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Amicus Therapeutics Inc
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Publication of PT2040548E publication Critical patent/PT2040548E/pt

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/185Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19Carboxylic acids, e.g. valproic acid
    • A61K31/194Carboxylic acids, e.g. valproic acid having two or more carboxyl groups, e.g. succinic, maleic or phthalic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/437Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • A61K31/45Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02Muscle relaxants, e.g. for tetanus or cramps
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A90/00Technologies having an indirect contribution to adaptation to climate change
    • Y02A90/10Information and communication technologies [ICT] supporting adaptation to climate change, e.g. for weather forecasting or climate simulation

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Pain & Pain Management (AREA)
  • Hospice & Palliative Care (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Hydrogenated Pyridines (AREA)
  • Saccharide Compounds (AREA)
  • Medicines Containing Plant Substances (AREA)
  • Steroid Compounds (AREA)
PT07812295T 2006-06-23 2007-06-25 Método para o tratamento de distúrbios neurológicos através do aumento da atividade da -glucocerebrosidase PT2040548E (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US81595206P 2006-06-23 2006-06-23

Publications (1)

Publication Number Publication Date
PT2040548E true PT2040548E (pt) 2012-08-02

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ID=38834440

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PT07812295T PT2040548E (pt) 2006-06-23 2007-06-25 Método para o tratamento de distúrbios neurológicos através do aumento da atividade da -glucocerebrosidase

Country Status (15)

Country Link
US (4) US7829579B2 (enExample)
EP (1) EP2040548B1 (enExample)
JP (1) JP5303458B2 (enExample)
KR (1) KR101402554B1 (enExample)
AT (1) ATE555788T1 (enExample)
AU (1) AU2007260812B2 (enExample)
BR (1) BRPI0713442A2 (enExample)
CA (1) CA2656643C (enExample)
DK (1) DK2040548T3 (enExample)
ES (1) ES2387845T3 (enExample)
IL (1) IL196151A (enExample)
MX (1) MX2009000032A (enExample)
PT (1) PT2040548E (enExample)
WO (1) WO2007150064A2 (enExample)
ZA (1) ZA200900270B (enExample)

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US9206457B2 (en) * 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
EP2435459B1 (en) * 2009-05-26 2016-05-04 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of acid beta-glucosidase
CN105126086B (zh) * 2009-07-28 2019-05-14 夏尔人类遗传性治疗公司 用于治疗戈谢病的组合物和方法
EP2490533B1 (en) 2009-10-19 2015-09-16 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
RU2015147509A (ru) 2009-10-19 2019-01-14 Амикус Терапьютикс, Инк. Новые композиции для предупреждения и/или лечения дегенеративных расстройств центральной нервной системы
WO2011049787A1 (en) * 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases
DK2646044T3 (da) 2010-11-30 2019-11-25 Orphazyme As Metoder til at øge den intracellulære aktivitet af Hsp70
WO2012154967A1 (en) 2011-05-12 2012-11-15 Proteostasis Therapeutics, Inc. Proteostasis regulators
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CN103764166B (zh) 2011-06-22 2017-10-24 通用医疗公司 蛋白质病的治疗
JP2015504919A (ja) 2012-01-25 2015-02-16 プロテオステイシス セラピューティクス,インコーポレイテッド プロテアソーム活性を調節する化合物
MA38144A1 (fr) * 2012-11-05 2018-08-31 Genzyme Corp Compositions et procédés pour le traitement de protéinopathies
US9750712B2 (en) 2012-12-07 2017-09-05 Rush University Medical Center Composition and method for treating neuronal ceroid lipofuscinosis
EP3087056A4 (en) 2013-12-23 2017-07-19 Alectos Therapeutics Inc. Glucocerebrosidase modulators and uses thereof
US9675627B2 (en) 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
KR20250069686A (ko) 2014-09-15 2025-05-19 제브라 덴마크 에이/에스 아리모클로몰 제제
PT3215511T (pt) 2014-11-06 2024-05-22 Bial R&D Invest S A Pirazolo(1,5-a)pirimidinas substituídas e a sua utilização no tratamento de distúrbios médicos
EP3292206B8 (en) 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
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Publication number Publication date
BRPI0713442A2 (pt) 2012-03-06
AU2007260812A1 (en) 2007-12-27
CA2656643A1 (en) 2007-12-27
KR101402554B1 (ko) 2014-06-19
ZA200900270B (en) 2011-02-23
US20170027919A1 (en) 2017-02-02
US20110052613A1 (en) 2011-03-03
JP5303458B2 (ja) 2013-10-02
DK2040548T3 (da) 2012-08-13
WO2007150064A2 (en) 2007-12-27
US7829579B2 (en) 2010-11-09
EP2040548A4 (en) 2009-12-23
CA2656643C (en) 2015-08-25
US20080009516A1 (en) 2008-01-10
WO2007150064A3 (en) 2008-02-21
KR20090021393A (ko) 2009-03-03
ATE555788T1 (de) 2012-05-15
US20150025109A1 (en) 2015-01-22
AU2007260812B2 (en) 2012-08-16
ES2387845T3 (es) 2012-10-02
JP2009541489A (ja) 2009-11-26
US10064851B2 (en) 2018-09-04
EP2040548A2 (en) 2009-04-01
IL196151A (en) 2015-08-31
US9119845B2 (en) 2015-09-01
MX2009000032A (es) 2009-01-23
US9402837B2 (en) 2016-08-02
EP2040548B1 (en) 2012-05-02
IL196151A0 (en) 2009-11-18

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