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MX2009011473A - Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas. - Google Patents

Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.

Info

Publication number
MX2009011473A
MX2009011473A MX2009011473A MX2009011473A MX2009011473A MX 2009011473 A MX2009011473 A MX 2009011473A MX 2009011473 A MX2009011473 A MX 2009011473A MX 2009011473 A MX2009011473 A MX 2009011473A MX 2009011473 A MX2009011473 A MX 2009011473A
Authority
MX
Mexico
Prior art keywords
dosing regimens
lysosomal storage
treatment
pharmacological chaperones
storage diseases
Prior art date
Application number
MX2009011473A
Other languages
English (en)
Inventor
Brandon Wustman
David Lockhart
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
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of MX2009011473A publication Critical patent/MX2009011473A/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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs 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
    • 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
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • 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)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Cardiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Psychiatry (AREA)
  • Obesity (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)

Abstract

La presente invención proporciona regímenes de dosificación para administrar chaperonas farmacológicas a un sujeto que lo necesite. Los regímenes de dosificación se pueden utilizar para tratar trastornos causados por un mal plegamiento de proteínas inadecuado, tales como trastornos por almacenamiento lisosomal.
MX2009011473A 2007-04-26 2008-04-28 Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas. MX2009011473A (es)

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

Publications (1)

Publication Number Publication Date
MX2009011473A true MX2009011473A (es) 2010-01-18

Family

ID=39926311

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2009011473A MX2009011473A (es) 2007-04-26 2008-04-28 Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.

Country Status (8)

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

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004069190A2 (en) 2003-01-31 2004-08-19 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiency disorders
EP2024745B1 (en) 2006-05-16 2015-07-08 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
SI2252313T1 (sl) 2008-02-12 2015-08-31 Amicus Therapeutics, Inc. Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
ES2963910T3 (es) 2008-06-26 2024-04-03 Zevra Denmark As Uso de Hsp70 como regulador de la actividad enzimática
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
CN102647905B (zh) 2009-10-19 2015-09-09 阿米库斯治疗学公司 用于预防和/或治疗溶酶体贮积失调的新颖组合物
PL2490712T3 (pl) 2009-10-19 2015-12-31 Amicus Therapeutics Inc Sposób leczenia choroby Alzheimera przy zastosowaniu chaperonów farmakologicznych dla zwiększenia aktywności gangliozydaz
KR101880411B1 (ko) 2009-10-19 2018-07-19 아미쿠스 세라퓨틱스, 인코포레이티드 중추신경계의 퇴행성 질환을 예방 및/또는 치료하는 신규 조성물
CA2778669C (en) * 2009-10-27 2019-04-16 Erytech Pharma Composition to induce specific immune tolerance
EP3028701B1 (en) * 2009-11-17 2019-11-13 Baylor Research Institute 1-deoxygalactonojirimycin for use in treating a cardiac disease
SI2796457T1 (sl) 2009-11-27 2016-10-28 Genzyme Corporation Genz 112638 za zdravljenje Gaucherjeve ali Fabryjeve bolezni v kombinirani terapiji
CA2817011C (en) * 2010-11-08 2019-04-02 Callidus Biopharma, Inc. Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity
PT2646044T (pt) 2010-11-30 2019-11-12 Orphazyme As Métodos para aumentar a atividade intracelular de hsp70
EA031874B1 (ru) 2011-03-11 2019-03-29 Амикус Терапьютикс, Инк. Способ лечения болезни фабри
EP2533051A1 (en) * 2011-06-06 2012-12-12 Centogene GmbH Method for the diagnosis of Gaucher's disease
KR20230066482A (ko) 2012-03-07 2023-05-15 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
ES2924829T3 (es) 2012-03-27 2022-10-11 Amicus Therapeutics Inc Compuestos novedosos para prevenir y/o tratar tesaurismosis lisosómicas y/o trastornos degenerativos del sistema nervioso central
HUE053565T2 (hu) * 2012-05-03 2021-07-28 Amicus Therapeutics Inc Adagolási rendek Pompe betegség kezelésére
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
WO2014014938A1 (en) * 2012-07-17 2014-01-23 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
US9675627B2 (en) 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
HUE054957T2 (hu) 2014-09-15 2021-10-28 Orphazyme As Arimoklomol készítése
MY186336A (en) 2014-09-30 2021-07-13 Amicus Therapeutics Inc Highly potent acid alpha-glucosidase with enhanced carbohydrates
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
US10179128B2 (en) * 2015-08-31 2019-01-15 Amicus Therapeutics, Inc. Regimens for treating and preventing lysosomal disorders and degenerative disorders of the central nervous system
WO2017041051A1 (en) 2015-09-04 2017-03-09 Sqz Biotechnologies Company Intracellular delivery of biomolecules to cells comprising a cell wall
CA3010205A1 (en) 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
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
MX2018011951A (es) 2016-03-30 2019-02-13 Amicus Therapeutics Inc Metodo para seleccionar proteinas recombinantes ricas en m6p.
IL301319A (en) 2016-03-30 2023-05-01 Amicus Therapeutics Inc Formulations that include recombinant acid alpha-glucosidase
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
RU2021117465A (ru) 2016-04-29 2021-07-22 Орфазим А/С Аримокломол для лечения ассоциированных с глюкоцереброзидазой нарушений
SG11201809437TA (en) 2016-05-03 2018-11-29 Sqz Biotechnologies Co 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.
JP7250698B2 (ja) 2017-04-25 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 中枢神経系の変性疾患及び／又はリソソーム蓄積疾患を予防及び／又は治療するための新規組成物
EP3624831B1 (en) 2017-05-15 2023-03-29 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
KR20240017111A (ko) 2017-05-30 2024-02-06 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
EP4374918B1 (en) 2017-05-30 2025-07-16 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
WO2019157047A1 (en) 2018-02-06 2019-08-15 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
CN113826015A (zh) * 2018-12-10 2021-12-21 戴纳立制药公司 溶酶体贮积症的生物标志物及其使用方法
MX2021015352A (es) * 2019-06-11 2022-04-06 Amicus Therapeutics Inc Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal.
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
IL303026A (en) 2020-11-19 2023-07-01 Zevra Denmark As 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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
WO2004069190A2 (en) * 2003-01-31 2004-08-19 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiency disorders
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
EP3441090A1 (en) * 2005-05-17 2019-02-13 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
MX2007015602A (es) * 2005-06-08 2008-02-21 Amicus Therapeutics Inc Tratamiento de trastornos del sistema nervioso central asociados con mutaciones en genes que codifican las enzimas lisosomicas.
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
EP2024745B1 (en) * 2006-05-16 2015-07-08 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
JP2010523578A (ja) 2007-03-30 2010-07-15 アミカス セラピューティックス インコーポレイテッド 薬理シャペロンを用いるファブリー病の治療方法

Also Published As

Publication number Publication date
US20150352093A1 (en) 2015-12-10
US9056101B2 (en) 2015-06-16
JP2010525084A (ja) 2010-07-22
AU2008245578A1 (en) 2008-11-06
US20100266571A1 (en) 2010-10-21
CA2685332A1 (en) 2008-11-06
WO2008134628A3 (en) 2010-01-14
WO2008134628A2 (en) 2008-11-06
EP2150254A2 (en) 2010-02-10
EP2150254A4 (en) 2010-11-10
IL201733A0 (en) 2010-06-16

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