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MX2022011334A - Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert. - Google Patents

Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert.

Info

Publication number
MX2022011334A
MX2022011334A MX2022011334A MX2022011334A MX2022011334A MX 2022011334 A MX2022011334 A MX 2022011334A MX 2022011334 A MX2022011334 A MX 2022011334A MX 2022011334 A MX2022011334 A MX 2022011334A MX 2022011334 A MX2022011334 A MX 2022011334A
Authority
MX
Mexico
Prior art keywords
ert
treatment
fabry disease
naã
experienced patients
Prior art date
Application number
MX2022011334A
Other languages
English (en)
Inventor
Jeff Castelli
Original Assignee
Amicus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from US15/213,920 external-priority patent/US9999618B2/en
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of MX2022011334A publication Critical patent/MX2022011334A/es

Links

Classifications

    • 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
    • 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
    • A61K31/45—Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7008—Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • A61P39/00—General protective or antinoxious agents
    • A61P39/02—Antidotes
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
  • Toxicology (AREA)
  • Molecular Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)

Abstract

Se proveen regímenes de dosificación para el tratamiento de la enfermedad de Fabry en un paciente. Algunos métodos se relacionan con el tratamiento de pacientes con Fabry no tratados previamente o tratados con ERT. Algunos métodos comprenden administrar al paciente aproximadamente 123 mg de equivalentes de base libre de migalastat para mejorar la masa ventricular izquierda y/o para mejorar la globotriaosilceramida en podocitos.
MX2022011334A 2016-07-19 2018-06-20 Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert. MX2022011334A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US15/213,920 US9999618B2 (en) 2007-04-26 2016-07-19 Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones

Publications (1)

Publication Number Publication Date
MX2022011334A true MX2022011334A (es) 2022-10-07

Family

ID=59416847

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018007669A MX2018007669A (es) 2016-07-19 2017-07-19 Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert.
MX2022011334A MX2022011334A (es) 2016-07-19 2018-06-20 Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MX2018007669A MX2018007669A (es) 2016-07-19 2017-07-19 Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert.

Country Status (14)

Country Link
US (2) US12070453B2 (es)
EP (5) EP4389213A3 (es)
JP (4) JP6752270B2 (es)
KR (3) KR20240017126A (es)
CN (1) CN109661230A (es)
AR (1) AR109103A1 (es)
AU (2) AU2017298376A1 (es)
CA (1) CA3031249A1 (es)
CL (1) CL2018001677A1 (es)
CO (1) CO2018006358A2 (es)
IL (2) IL313907A (es)
MX (2) MX2018007669A (es)
TW (1) TW202408513A (es)
WO (1) WO2018017721A1 (es)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2533050B3 (en) 2006-05-16 2015-06-24 Amicus Therapeutics, Inc. Treatment Options For Fabry Disease
AU2009214648B2 (en) 2008-02-12 2014-11-13 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
EP4389213A3 (en) * 2016-07-19 2024-08-21 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naive and ert-experienced patients
US12020820B1 (en) * 2017-03-03 2024-06-25 Cerner Innovation, Inc. Predicting sphingolipidoses (fabry's disease) and decision support
DK4062916T3 (da) 2017-05-30 2025-01-13 Amicus Therapeutics Inc Behandling af fabry-patienter med specifikke mutationer
WO2018222655A1 (en) 2017-05-30 2018-12-06 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
CA3090496C (en) * 2018-02-06 2024-03-26 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
LT3749308T (lt) * 2018-02-06 2024-02-12 Amicus Therapeutics, Inc. Klasikine fabri liga sergančių pacientų gydymas migalastatu
AU2019326252A1 (en) * 2018-08-20 2021-02-25 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the GLA gene
US20220313670A1 (en) * 2019-06-11 2022-10-06 Amicus Therapeutics, Inc. Methods Of Treating Fabry Disease In Patients Having Renal Impairment
BR112022002202A2 (pt) 2019-08-07 2022-06-14 Amicus Therapeutics Inc Métodos de tratar doença de fabry em pacientes tendo uma mutação no gene gla
JP2023513700A (ja) * 2020-02-10 2023-04-03 アミカス セラピューティックス インコーポレイテッド ファブリー病の治療方法
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
KR102471225B1 (ko) 2021-11-18 2022-11-28 박기용 어플리케이션을 통한 소비자 및 농장 연결 시스템

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0463109A4 (en) 1989-03-24 1992-11-19 Research Corporation Technologies, Inc. Recombinant alpha-galactosidase, a therapy for fabry disease
EP0536402A4 (en) 1990-06-29 1993-05-12 Nippon Shinyaku Company, Limited Piperidine derivative
JP2000509971A (ja) 1996-04-10 2000-08-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 化学的シャペローンを用いる遺伝的欠陥の修正
US6270954B1 (en) 1996-04-10 2001-08-07 The Regents Of The University Of California Correction of genetic defects using chemical chaperones
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
SI2444102T1 (sl) 2003-01-31 2015-08-31 Mount Sinai School Of Medicine Of New York University Kombinirana terapija za zdravljenje motenj pomanjkanja proteina
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
US7741340B2 (en) 2003-11-12 2010-06-22 Amicus Therapeutics, Inc. Hydroxy piperidine derivatives to treat gaucher disease
AU2006247065B8 (en) 2005-05-17 2012-07-12 Amicus Therapeutics, Inc. A method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives
CN104771402A (zh) 2005-06-08 2015-07-15 阿米库斯治疗学公司 溶酶体酶编码基因突变相关的cns紊乱的治疗
US7955262B2 (en) 2005-07-26 2011-06-07 Syneron Medical Ltd. Method and apparatus for treatment of skin using RF and ultrasound energies
JP2009502954A (ja) 2005-07-27 2009-01-29 ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド タンパク質のミスフォールディングを修正する小分子及びその使用
EP2533050B3 (en) * 2006-05-16 2015-06-24 Amicus Therapeutics, Inc. Treatment Options For Fabry Disease
AU2015230773B2 (en) * 2006-05-16 2017-09-28 Amicus Therapeutics Inc. Assays for diagnosing and evaluating treatment options for fabry disease
EP2081904B1 (en) 2006-09-22 2011-03-02 Krka Tovarna Zdravil, D.D., Novo Mesto Aripiprazole hemifumarate and process for its preparation
WO2008121826A2 (en) 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones
AU2009214648B2 (en) 2008-02-12 2014-11-13 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
SG10201604757RA (en) * 2011-03-11 2016-08-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
JP2013255488A (ja) * 2012-05-18 2013-12-26 Saitama Medical Univ 拡張期心不全非ヒトモデル動物及びその製造方法、並びに、拡張期心不全予防乃至症状改善活性を有する物質のスクリーニング方法
EP4389213A3 (en) * 2016-07-19 2024-08-21 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naive and ert-experienced patients

Also Published As

Publication number Publication date
EP4389213A2 (en) 2024-06-26
WO2018017721A1 (en) 2018-01-25
US20230321065A1 (en) 2023-10-12
TW201806597A (zh) 2018-03-01
KR20230061563A (ko) 2023-05-08
AU2023206175A1 (en) 2023-08-10
EP4324521A2 (en) 2024-02-21
US20190183869A1 (en) 2019-06-20
CA3031249A1 (en) 2018-01-25
JP2019178140A (ja) 2019-10-17
KR20240017126A (ko) 2024-02-06
JP6752270B2 (ja) 2020-09-09
JP2022033749A (ja) 2022-03-02
AR109103A1 (es) 2018-10-31
KR20190030729A (ko) 2019-03-22
IL313907A (en) 2024-08-01
CN109661230A (zh) 2019-04-19
TW202408513A (zh) 2024-03-01
US12070453B2 (en) 2024-08-27
JP2018526368A (ja) 2018-09-13
EP3487502A1 (en) 2019-05-29
CO2018006358A2 (es) 2018-11-30
EP4356911A1 (en) 2024-04-24
EP4389213A3 (en) 2024-08-21
MX2018007669A (es) 2018-09-12
EP4324521A3 (en) 2024-04-24
EP4378524A2 (en) 2024-06-05
AU2017298376A1 (en) 2019-03-07
IL264301A (en) 2019-02-28
CL2018001677A1 (es) 2019-02-01
JP2024026079A (ja) 2024-02-28
EP4378524A3 (en) 2024-08-14

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