JP2010525084A - 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画 - Google Patents

薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画 Download PDF

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JP2010525084A
JP2010525084A JP2010506557A JP2010506557A JP2010525084A JP 2010525084 A JP2010525084 A JP 2010525084A JP 2010506557 A JP2010506557 A JP 2010506557A JP 2010506557 A JP2010506557 A JP 2010506557A JP 2010525084 A JP2010525084 A JP 2010525084A
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JP2010525084A5 (OSRAM
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デイビッド・ロックハート
ブランドン・ウストマン
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アミカス セラピューティックス インコーポレイテッド
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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
    • A61K31/33Heterocyclic 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
    • 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
    • A61P3/00Drugs for disorders of the metabolism
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system

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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)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Hospice & Palliative Care (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Hydrogenated Pyridines (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2010506557A 2007-04-26 2008-04-28 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画 Pending JP2010525084A (ja)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US91428807P 2007-04-26 2007-04-26
US1474407P 2007-12-18 2007-12-18
US2810508P 2008-02-12 2008-02-12
PCT/US2008/061764 WO2008134628A2 (en) 2007-04-26 2008-04-28 Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones

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JP2010525084A true JP2010525084A (ja) 2010-07-22
JP2010525084A5 JP2010525084A5 (OSRAM) 2011-05-19

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US (2) US9056101B2 (OSRAM)
EP (1) EP2150254A4 (OSRAM)
JP (1) JP2010525084A (OSRAM)
AU (1) AU2008245578A1 (OSRAM)
CA (1) CA2685332A1 (OSRAM)
IL (1) IL201733A0 (OSRAM)
MX (1) MX2009011473A (OSRAM)
WO (1) WO2008134628A2 (OSRAM)

Cited By (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015519329A (ja) * 2012-05-03 2015-07-09 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための投与計画
JP2017511372A (ja) * 2014-04-14 2017-04-20 アミカス セラピューティックス インコーポレイテッド 脳アミロイドーシスを処置及び/又は予防する投与計画
JP2017161528A (ja) * 2011-06-06 2017-09-14 セントジーン アーゲー ゴーシェ病の診断のための方法
JP2018528953A (ja) * 2015-08-31 2018-10-04 アミカス セラピューティックス インコーポレイテッド リソソーム障害及び中枢神経系の変性障害の治療及び予防のための(3r,4r,5s)−5−(ジフルオロメチル)ピペリジン−3,4−ジオールを含むレジメン
US10208299B2 (en) 2014-09-30 2019-02-19 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
US10227577B2 (en) 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
US10512676B2 (en) 2016-03-30 2019-12-24 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
JP2020507562A (ja) * 2017-05-30 2020-03-12 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法
US10857212B2 (en) 2015-12-30 2020-12-08 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of Pompe disease
US12246062B2 (en) 2017-05-15 2025-03-11 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase

Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT2444102E (pt) 2003-01-31 2015-09-17 Sinai School Medicine Terapia combinada para o tratamento de distúrbios de deficiência proteica
FI3457135T3 (fi) 2006-05-16 2025-03-08 Amicus Therapeutics Inc Fabryn taudin hoitovaihtoehtoja
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
HUE026543T2 (hu) 2008-02-12 2016-06-28 Amicus Therapeutics Inc Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére
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WO2010048532A1 (en) 2008-10-24 2010-04-29 Amicus Therapeutics, Inc. Multiple compartment dosing model
WO2010056746A1 (en) * 2008-11-11 2010-05-20 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
GB0906159D0 (en) * 2009-04-09 2009-05-20 Summit Corp Plc Drug combination for the treatment of proteostatic diseases
CA2758271C (en) * 2009-04-09 2018-06-05 Robert Boyd Methods for preventing and/or treating lysosomal storage disorders
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
RU2015147509A (ru) 2009-10-19 2019-01-14 Амикус Терапьютикс, Инк. Новые композиции для предупреждения и/или лечения дегенеративных расстройств центральной нервной системы
EP2490533B1 (en) 2009-10-19 2015-09-16 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
PT2493487T (pt) * 2009-10-27 2016-11-08 Erytech Pharma Composição para induzir uma tolerância imunitária específica
US9066939B2 (en) * 2009-11-17 2015-06-30 Baylor Research Institute Urinary triaosylceramide (GB3) as a marker of cardiac disease
ES2754398T3 (es) 2009-11-27 2020-04-17 Genzyme Corp Eliglustat (Genz 112638) como inhibidor de glucosilceramida-sintasa para uso en un método de tratamiento de la enfermedad de fabry o de gaucher, comprendiendo el método ajustar la dosis terapéutica individual al metabolismo P-450 del paciente
US8962564B2 (en) 2010-11-08 2015-02-24 Amicus Therapeutics, Inc. Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity
DK2646044T3 (da) 2010-11-30 2019-11-25 Orphazyme As Metoder til at øge den intracellulære aktivitet af Hsp70
SG10201604757RA (en) * 2011-03-11 2016-08-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
KR20140135222A (ko) 2012-03-07 2014-11-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
HK1205503A1 (en) 2012-03-27 2015-12-18 Amicus Therapeutics, Inc. Novel compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system
US10155027B2 (en) 2012-07-17 2018-12-18 Amicus Therapeutics, Inc. Alpha-galactosidase A and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease
CA2917995C (en) 2012-07-17 2021-01-26 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
KR20250069686A (ko) 2014-09-15 2025-05-19 제브라 덴마크 에이/에스 아리모클로몰 제제
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
US11613759B2 (en) 2015-09-04 2023-03-28 Sqz Biotechnologies Company Intracellular delivery of biomolecules to cells comprising a cell wall
JP6438421B2 (ja) * 2016-02-17 2018-12-12 ファナック株式会社 電動機のステータ
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
LT3448382T (lt) 2016-04-29 2021-04-12 Orphazyme A/S Arimoklomolis, skirtas su gliukocerebrozidaze susijusiems sutrikimams gydyti
AU2017259987B2 (en) 2016-05-03 2023-10-19 Sqz Biotechnologies Company Intracellular delivery of biomolecules to induce tolerance
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
CN110573492A (zh) 2017-04-25 2019-12-13 阿米库斯治疗学公司 用于预防和/或治疗中枢神经系统退行性失调和/或溶酶体贮积失调的新颖组合物
TWI795408B (zh) 2017-05-30 2023-03-11 美商阿米庫斯醫療股份有限公司 治療有腎損傷的法布里患者的方法
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CA3121927A1 (en) * 2018-12-10 2020-06-18 Denali Therapeutics Inc. Lysosomal storage disorder biomarkers and methods of use thereof
AR120055A1 (es) * 2019-06-11 2022-02-02 Amicus Therapeutics Inc Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal
CA3147055A1 (en) 2019-08-07 2021-02-11 Elfrida Benjamin Methods of treating fabry disease in patients having a mutation in the gla gene
US20230416224A1 (en) 2020-11-19 2023-12-28 Zevra Denmark A/S Processes for preparing arimoclomol citrate and intermediates thereof
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2003528799A (ja) * 1998-06-01 2003-09-30 マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー リソソームα−ガラクトシダーゼAの増強方法
JP2006516645A (ja) * 2003-01-31 2006-07-06 マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー タンパク質欠損性障害の治療のための併用療法
WO2006125141A2 (en) * 2005-05-17 2006-11-23 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin derivatives
WO2006133446A2 (en) * 2005-06-08 2006-12-14 Amicus Therapeutics, Inc. Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7446098B2 (en) * 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
US7955262B2 (en) 2005-07-26 2011-06-07 Syneron Medical Ltd. Method and apparatus for treatment of skin using RF and ultrasound energies
AU2006272497B2 (en) * 2005-07-27 2012-07-19 University Of Florida Research Foundation, Inc. Small compounds that correct protein misfolding and uses thereof
FI3457135T3 (fi) * 2006-05-16 2025-03-08 Amicus Therapeutics Inc Fabryn taudin hoitovaihtoehtoja
WO2008121826A2 (en) * 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2003528799A (ja) * 1998-06-01 2003-09-30 マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー リソソームα−ガラクトシダーゼAの増強方法
JP2006516645A (ja) * 2003-01-31 2006-07-06 マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー タンパク質欠損性障害の治療のための併用療法
WO2006125141A2 (en) * 2005-05-17 2006-11-23 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin derivatives
WO2006133446A2 (en) * 2005-06-08 2006-12-14 Amicus Therapeutics, Inc. Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
岡野定輔, 新.薬剤学総論, vol. 改訂第3版, JPN6013011899, 1987, pages 240 - 248, ISSN: 0002481224 *

Cited By (28)

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JP2017161528A (ja) * 2011-06-06 2017-09-14 セントジーン アーゲー ゴーシェ病の診断のための方法
JP2020045346A (ja) * 2012-05-03 2020-03-26 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための投与計画
JP2018109003A (ja) * 2012-05-03 2018-07-12 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための投与計画
JP7046241B2 (ja) 2012-05-03 2022-04-01 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための投与計画
JP2015519329A (ja) * 2012-05-03 2015-07-09 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための投与計画
JP2021088561A (ja) * 2012-05-03 2021-06-10 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための投与計画
JP2017511372A (ja) * 2014-04-14 2017-04-20 アミカス セラピューティックス インコーポレイテッド 脳アミロイドーシスを処置及び/又は予防する投与計画
US11591583B2 (en) 2014-09-30 2023-02-28 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
US11753632B2 (en) 2014-09-30 2023-09-12 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
US10208299B2 (en) 2014-09-30 2019-02-19 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
US10961522B2 (en) 2014-09-30 2021-03-30 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
JP2018528953A (ja) * 2015-08-31 2018-10-04 アミカス セラピューティックス インコーポレイテッド リソソーム障害及び中枢神経系の変性障害の治療及び予防のための(3r,4r,5s)−5−(ジフルオロメチル)ピペリジン−3,4−ジオールを含むレジメン
US12414985B2 (en) 2015-12-30 2025-09-16 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of Pompe disease
US10857212B2 (en) 2015-12-30 2020-12-08 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of Pompe disease
US11278601B2 (en) 2015-12-30 2022-03-22 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of Pompe disease
US11491211B2 (en) 2016-03-30 2022-11-08 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
US11441138B2 (en) 2016-03-30 2022-09-13 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
US10512676B2 (en) 2016-03-30 2019-12-24 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
US10227577B2 (en) 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
US12246062B2 (en) 2017-05-15 2025-03-11 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
KR20210066032A (ko) * 2017-05-30 2021-06-04 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
JP2020203886A (ja) * 2017-05-30 2020-12-24 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法
JP2022071094A (ja) * 2017-05-30 2022-05-13 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法
KR102427610B1 (ko) 2017-05-30 2022-07-29 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
JP2020073500A (ja) * 2017-05-30 2020-05-14 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法
JP2020507562A (ja) * 2017-05-30 2020-03-12 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法
JP2023052005A (ja) * 2017-05-30 2023-04-11 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法
JP2023100658A (ja) * 2017-05-30 2023-07-19 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法

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WO2008134628A2 (en) 2008-11-06
US9056101B2 (en) 2015-06-16
AU2008245578A1 (en) 2008-11-06
MX2009011473A (es) 2010-01-18
EP2150254A4 (en) 2010-11-10
WO2008134628A3 (en) 2010-01-14
EP2150254A2 (en) 2010-02-10
CA2685332A1 (en) 2008-11-06
US20100266571A1 (en) 2010-10-21
US20150352093A1 (en) 2015-12-10
IL201733A0 (en) 2010-06-16

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