KR20140011367A - 파브리 병을 치료하기 위한 투여 요법 - Google Patents

파브리 병을 치료하기 위한 투여 요법 Download PDF

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Publication number
KR20140011367A
KR20140011367A KR1020137026394A KR20137026394A KR20140011367A KR 20140011367 A KR20140011367 A KR 20140011367A KR 1020137026394 A KR1020137026394 A KR 1020137026394A KR 20137026394 A KR20137026394 A KR 20137026394A KR 20140011367 A KR20140011367 A KR 20140011367A
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KR
South Korea
Prior art keywords
gal
agalsidase
administration
deoxygalactonozirimycin
patients
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Ceased
Application number
KR1020137026394A
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English (en)
Korean (ko)
Inventor
더글라스 스튜어트 그린
케네쓰 조셉 발렌자노
Original Assignee
아미쿠스 세라퓨틱스, 인코포레이티드
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=46831259&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=KR20140011367(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 아미쿠스 세라퓨틱스, 인코포레이티드 filed Critical 아미쿠스 세라퓨틱스, 인코포레이티드
Publication of KR20140011367A publication Critical patent/KR20140011367A/ko
Ceased legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic 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
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01062—Glycosylceramidase (3.2.1.62)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Cardiology (AREA)
  • Genetics & Genomics (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Diabetes (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Hydrogenated Pyridines (AREA)
KR1020137026394A 2011-03-11 2012-03-08 파브리 병을 치료하기 위한 투여 요법 Ceased KR20140011367A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201161451798P 2011-03-11 2011-03-11
US61/451,798 2011-03-11
US201161578201P 2011-12-20 2011-12-20
US61/578,201 2011-12-20
US201261596165P 2012-02-07 2012-02-07
US61/596,165 2012-02-07
PCT/US2012/028260 WO2012125402A2 (en) 2011-03-11 2012-03-08 Dosing regimens for the treatment of fabry disease

Publications (1)

Publication Number Publication Date
KR20140011367A true KR20140011367A (ko) 2014-01-28

Family

ID=46831259

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020137026394A Ceased KR20140011367A (ko) 2011-03-11 2012-03-08 파브리 병을 치료하기 위한 투여 요법

Country Status (16)

Country Link
US (4) US20140219986A1 (enExample)
EP (2) EP2683382B1 (enExample)
JP (6) JP2014528901A (enExample)
KR (1) KR20140011367A (enExample)
CN (2) CN107088225A (enExample)
AU (6) AU2012229330B2 (enExample)
CA (1) CA2829947C (enExample)
CL (1) CL2013002601A1 (enExample)
EA (1) EA031874B1 (enExample)
ES (1) ES2807502T3 (enExample)
HK (1) HK1242998A1 (enExample)
MX (1) MX2013010446A (enExample)
SG (2) SG10201604757RA (enExample)
TW (3) TWI624259B (enExample)
WO (1) WO2012125402A2 (enExample)
ZA (1) ZA201306735B (enExample)

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JP2009537149A (ja) 2006-05-16 2009-10-29 アミークス セラピューティックス インコーポレイテッド ファブリー病に対する治療の選択を診断し、評価するためのアッセイ
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
UY34317A (es) 2011-09-12 2013-02-28 Genzyme Corp Anticuerpo antireceptor de célula T (alfa)/ß
US9790268B2 (en) 2012-09-12 2017-10-17 Genzyme Corporation Fc containing polypeptides with altered glycosylation and reduced effector function
RU2711935C2 (ru) 2013-03-11 2020-01-23 Джензим Корпорейшн Сайт-специфическая конъюгация антитело-лекарственное средство посредством гликоинженерии
EP3108002A1 (en) * 2014-02-17 2016-12-28 GlaxoSmithKline Intellectual Property Development Limited Microbiological process
PL3129067T3 (pl) 2014-03-19 2023-05-08 Genzyme Corporation Specyficzne dla miejsca glikomodyfikowanie ugrupowań celujących
US9675627B2 (en) * 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
GB201508025D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
CN108351862B (zh) 2015-08-11 2023-08-22 科格诺亚公司 利用人工智能和用户输入来确定发育进展的方法和装置
US11972336B2 (en) 2015-12-18 2024-04-30 Cognoa, Inc. Machine learning platform and system for data analysis
KR20190087673A (ko) * 2016-03-22 2019-07-24 아미쿠스 세라퓨틱스, 인코포레이티드 Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
AU2017298376A1 (en) * 2016-07-19 2019-03-07 Amicus Therapeutics, Inc. Treatment of Fabry disease in ERT-naive and ERT-experienced patients
WO2018090009A1 (en) 2016-11-14 2018-05-17 Cognoa, Inc. Methods and apparatus for evaluating developmental conditions and providing control over coverage and reliability
WO2018148365A1 (en) * 2017-02-09 2018-08-16 Cognoa, Inc. Platform and system for digital personalized medicine
PT4324522T (pt) * 2017-05-30 2026-03-13 Amicus Therapeutics Inc Métodos de tratamento de pacientes com fabry tendo insuficiência renal
KR20220116305A (ko) 2017-05-30 2022-08-22 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
JP7066157B2 (ja) 2017-06-14 2022-05-13 学校法人 明治薬科大学 ファブリー病治療用医薬の組合せ物及びその利用
EP3749307B1 (en) 2018-02-06 2025-12-10 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
EP4289476A3 (en) * 2018-08-20 2024-02-21 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
AU2020248351B2 (en) 2019-03-22 2025-09-25 Cognoa, Inc. Personalized digital therapy methods and devices
MA55529A (fr) 2019-04-03 2022-02-09 Genzyme Corp Polypeptides de liaison anti-alpha bêta tcr à fragmentation réduite
BR112021024886A2 (pt) * 2019-06-11 2022-01-25 Amicus Therapeutics Inc Métodos de tratar a doença de fabry em pacientes com insuficiência renal
WO2021026447A1 (en) 2019-08-07 2021-02-11 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
CA3153086A1 (en) 2019-09-06 2021-03-11 Cognoa, Inc. Methods, systems, and devices for the diagnosis of behavioral disorders, developmental delays, and neurologic impairments
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
US20240120050A1 (en) * 2022-10-07 2024-04-11 Insight Direct Usa, Inc. Machine learning method for predicting a health outcome of a patient using video and audio analytics
US20240197706A1 (en) * 2022-12-13 2024-06-20 Amicus Therapeutics, Inc. Methods of improving the pharmacokinetics of migalastat
TR2024011149A1 (tr) * 2024-08-23 2026-03-23 Sanovel İlaç Sanayi̇ Ve Ti̇caret Anoni̇m Şi̇rketi̇ Mi̇galastat i̇çeren kapsül formülasyonlari
TR2024011147A1 (tr) 2024-08-23 2026-03-23 Sanovel İlaç Sanayi̇ Ve Ti̇caret Anoni̇m Şi̇rketi̇ Mi̇galastat i̇çeren kapsül formülasyonu

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4551177A (en) * 1984-04-23 1985-11-05 National Starch And Chemical Corporation Compressible starches as binders for tablets or capsules
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
PT2857036T (pt) * 2003-01-31 2017-03-13 Mount Sinai School Of Medicine Of N Y Univ Terapia combinada para o tratamento de distúrbios de deficiência proteica
US7879354B2 (en) * 2004-01-13 2011-02-01 Mcneil-Ppc, Inc. Rapidly disintegrating gelatinous coated tablets
CN100457106C (zh) * 2005-06-15 2009-02-04 同济大学 1-脱氧野尻霉素在制备治疗糖尿病肾病药物中的应用
JP2009537149A (ja) * 2006-05-16 2009-10-29 アミークス セラピューティックス インコーポレイテッド ファブリー病に対する治療の選択を診断し、評価するためのアッセイ
MX2009010557A (es) * 2007-03-30 2009-11-19 Amicus Therapeutics Inc Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas.
WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
US20100291060A1 (en) * 2007-08-29 2010-11-18 Shire Human Genetic Therapies, Inc Subcutaneous administration of alpha-galactosidase a

Also Published As

Publication number Publication date
AU2016204445B2 (en) 2018-03-22
JP2021098697A (ja) 2021-07-01
JP2026035578A (ja) 2026-03-04
EA031874B1 (ru) 2019-03-29
TW201302197A (zh) 2013-01-16
US20200268890A1 (en) 2020-08-27
ZA201306735B (en) 2014-07-30
EP3698792A1 (en) 2020-08-26
JP2020033360A (ja) 2020-03-05
AU2018201637A1 (en) 2018-04-05
TW201740945A (zh) 2017-12-01
WO2012125402A3 (en) 2014-05-01
SG10201604757RA (en) 2016-08-30
CN107088225A (zh) 2017-08-25
AU2022203011A1 (en) 2022-05-26
CA2829947A1 (en) 2012-09-20
ES2807502T3 (es) 2021-02-23
EP2683382A2 (en) 2014-01-15
AU2024205251A1 (en) 2024-08-22
TWI624258B (zh) 2018-05-21
CN103974619A (zh) 2014-08-06
CN103974619B (zh) 2017-02-15
EP2683382B1 (en) 2020-04-22
NZ615726A (en) 2015-01-30
US20140219986A1 (en) 2014-08-07
HK1242998A1 (zh) 2018-07-06
CL2013002601A1 (es) 2014-03-28
JP2014528901A (ja) 2014-10-30
SG193379A1 (en) 2013-10-30
AU2020201715A1 (en) 2020-04-09
JP2023093448A (ja) 2023-07-04
TW201634048A (zh) 2016-10-01
AU2016204445A1 (en) 2016-07-21
JP2017132780A (ja) 2017-08-03
EP2683382A4 (en) 2014-10-22
AU2012229330A1 (en) 2013-10-03
AU2012229330B2 (en) 2016-04-21
WO2012125402A2 (en) 2012-09-20
MX2013010446A (es) 2014-03-27
TWI624259B (zh) 2018-05-21
EA201301018A1 (ru) 2014-04-30
US20240115708A1 (en) 2024-04-11
US20180153999A1 (en) 2018-06-07
CA2829947C (en) 2019-01-15

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