HK1242998A1 - 治疗法布里病的给药方案 - Google Patents

治疗法布里病的给药方案 Download PDF

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Publication number
HK1242998A1
HK1242998A1 HK18102615.1A HK18102615A HK1242998A1 HK 1242998 A1 HK1242998 A1 HK 1242998A1 HK 18102615 A HK18102615 A HK 18102615A HK 1242998 A1 HK1242998 A1 HK 1242998A1
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HK
Hong Kong
Prior art keywords
gal
enzyme
acase
migalastat
activity
Prior art date
Application number
HK18102615.1A
Other languages
English (en)
Chinese (zh)
Inventor
D.S.格林
K.J.瓦伦扎诺
K．J．瓦倫扎諾
Original Assignee
阿米库斯治疗学公司
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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=HK1242998(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 阿米库斯治疗学公司 filed Critical 阿米库斯治疗学公司
Publication of HK1242998A1 publication Critical patent/HK1242998A1/zh

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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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Cardiology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (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)
HK18102615.1A 2011-03-11 2018-02-23 治疗法布里病的给药方案 HK1242998A1 (zh)

Applications Claiming Priority (6)

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

Publications (1)

Publication Number Publication Date
HK1242998A1 true HK1242998A1 (zh) 2018-07-06

Family

ID=46831259

Family Applications (1)

Application Number Title Priority Date Filing Date
HK18102615.1A HK1242998A1 (zh) 2011-03-11 2018-02-23 治疗法布里病的给药方案

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) TW201740945A (enExample)
WO (1) WO2012125402A2 (enExample)
ZA (1) ZA201306735B (enExample)

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MX351004B (es) 2006-05-16 2017-09-26 Nat Institutes Of Health Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry.
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
US9580511B2 (en) 2013-03-11 2017-02-28 Genzyme Corporation Site-specific antibody-drug conjugation through glycoengineering
CN106687596A (zh) * 2014-02-17 2017-05-17 葛兰素史密斯克莱知识产权发展有限公司 微生物学方法
HUE061339T2 (hu) 2014-03-19 2023-06-28 Genzyme Corp Targetáló csoportok helyspecifikus glikotechnikai átalakítása
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
ES3067417T3 (en) 2015-08-11 2026-05-21 Cognoa Inc Apparatus to determine developmental progress with artificial intelligence and user input
US11972336B2 (en) 2015-12-18 2024-04-30 Cognoa, Inc. Machine learning platform and system for data analysis
EP3432882B1 (en) * 2016-03-22 2021-09-01 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having the g9331a mutation in the gla gene
WO2018017721A1 (en) * 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naïve and ert-experienced patients
CN118609834A (zh) 2016-11-14 2024-09-06 科格诺亚公司 用于评估发育状况并提供覆盖度和可靠性控制的方法和装置
JP7324709B2 (ja) * 2017-02-09 2023-08-10 コグノア，インク． デジタル個別化医療のためのプラットフォームとシステム
AU2018277756A1 (en) 2017-05-30 2020-01-23 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
PL4062916T3 (pl) * 2017-05-30 2025-03-17 Amicus Therapeutics, Inc. Leczenie pacjentów z chorobą Fabry’ego mających pewne mutacje
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
EP4717319A3 (en) 2018-02-06 2026-06-10 Amicus Therapeutics, Inc. Methods of treating fabry disease in pregnant patients
LT3840753T (lt) 2018-08-20 2024-11-11 Amicus Therapeutics, Inc. Migalastatas skirtas naudoti fabry ligos gydymo būdui, pacientams turintiems gla geno mutaciją
CN113873935B (zh) 2019-03-22 2024-11-22 科格诺亚公司 个性化数字化治疗方法和装置
JP7575401B2 (ja) 2019-04-03 2024-10-29 ジェンザイム・コーポレーション 断片化が低減した抗アルファベータｔｃｒ結合ポリペプチド
US20220313670A1 (en) * 2019-06-11 2022-10-06 Amicus Therapeutics, Inc. Methods Of Treating Fabry Disease In Patients Having Renal Impairment
KR20220044560A (ko) 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
WO2021046412A1 (en) 2019-09-06 2021-03-11 Cognoa, Inc. Methods, systems, and devices for the diagnosis of behavioral disorders, developmental delays, and neurologic impairments
FI4114390T3 (fi) * 2020-03-06 2025-03-07 Amicus Therapeutics Inc Menetelmiä fabryn taudin hoitamiseksi potilaissa, joilla on mutaatio gla-geenissä
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
CN118488839A (zh) * 2022-12-13 2024-08-13 阿米库斯治疗学公司 改善米加司他的药代动力学的方法
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 Ilac Sanayi Ve Ticaret Anonim Sirketi Mi̇galastat i̇çeren kapsül formülasyonlari
TR2024011147A1 (tr) * 2024-08-23 2026-03-23 Sanovel Ilac Sanayi Ve Ticaret Anonim Sirketi Mi̇galastat i̇çeren kapsül formülasyonu
US20260188467A1 (en) * 2024-12-27 2026-07-02 Center for Social Dynamics, LLC Parallel Processing Systems and Methods For Real-Time Multimodal Behavioral Classification Using Neural Networks With Dynamic Content Adaptation

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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
JP5589165B2 (ja) * 2003-01-31 2014-09-17 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
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
MX351004B (es) * 2006-05-16 2017-09-26 Nat Institutes Of Health Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry.
EP2142197A4 (en) * 2007-03-30 2010-11-10 Amicus Therapeutics Inc METHOD FOR THE TREATMENT OF MORBUS FABRY WITH PHARMACOLOGICAL CHAPTERONES
AU2008245578A1 (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
TWI624258B (zh) 2018-05-21
AU2018201637A1 (en) 2018-04-05
AU2012229330B2 (en) 2016-04-21
AU2020201715A1 (en) 2020-04-09
CA2829947C (en) 2019-01-15
CA2829947A1 (en) 2012-09-20
EP2683382B1 (en) 2020-04-22
SG10201604757RA (en) 2016-08-30
AU2012229330A1 (en) 2013-10-03
US20200268890A1 (en) 2020-08-27
CN103974619B (zh) 2017-02-15
JP2014528901A (ja) 2014-10-30
TW201634048A (zh) 2016-10-01
NZ615726A (en) 2015-01-30
US20140219986A1 (en) 2014-08-07
CL2013002601A1 (es) 2014-03-28
KR20140011367A (ko) 2014-01-28
SG193379A1 (en) 2013-10-30
ZA201306735B (en) 2014-07-30
CN103974619A (zh) 2014-08-06
US20180153999A1 (en) 2018-06-07
JP2021098697A (ja) 2021-07-01
EP2683382A4 (en) 2014-10-22
US20240115708A1 (en) 2024-04-11
JP2026035578A (ja) 2026-03-04
JP2023093448A (ja) 2023-07-04
WO2012125402A3 (en) 2014-05-01
TW201302197A (zh) 2013-01-16
EA201301018A1 (ru) 2014-04-30
EP3698792A1 (en) 2020-08-26
JP2020033360A (ja) 2020-03-05
AU2024205251A1 (en) 2024-08-22
TWI624259B (zh) 2018-05-21
AU2016204445A1 (en) 2016-07-21
JP2017132780A (ja) 2017-08-03
ES2807502T3 (es) 2021-02-23
AU2016204445B2 (en) 2018-03-22
CN107088225A (zh) 2017-08-25
EP2683382A2 (en) 2014-01-15
WO2012125402A2 (en) 2012-09-20
AU2022203011A1 (en) 2022-05-26
MX2013010446A (es) 2014-03-27
EA031874B1 (ru) 2019-03-29
TW201740945A (zh) 2017-12-01

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