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)
Spanish (es)
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)
  • Animal Behavior & Ethology (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Cardiology (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)
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 (cg-RX-API-DMAC7.html)
EP (1) EP2150254A4 (cg-RX-API-DMAC7.html)
JP (1) JP2010525084A (cg-RX-API-DMAC7.html)
AU (1) AU2008245578A1 (cg-RX-API-DMAC7.html)
CA (1) CA2685332A1 (cg-RX-API-DMAC7.html)
IL (1) IL201733A0 (cg-RX-API-DMAC7.html)
MX (1) MX2009011473A (cg-RX-API-DMAC7.html)
WO (1) WO2008134628A2 (cg-RX-API-DMAC7.html)

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EP2444102B1 (en) 2003-01-31 2015-04-08 The Mount Sinai School of Medicine of New York University Combination therapy for treating protein deficiency disorders
PL2533050T6 (pl) 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
EP2252313B1 (en) 2008-02-12 2015-04-08 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
WO2009155936A1 (en) 2008-06-26 2009-12-30 Orphazyme Aps Use of hsp70 as a regulator of enzymatic activity
US8321148B2 (en) 2008-10-24 2012-11-27 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
ES2564093T3 (es) 2009-04-09 2016-03-17 Amicus Therapeutics, Inc. Métodos para prevenir y/o tratar trastornos del almacenamiento lisosomal
ES2646099T3 (es) * 2009-10-19 2017-12-12 Amicus Therapeutics, Inc. Método para el tratamiento de la enfermedad de Alzheimer utilizando chaperonas farmacológicas para aumentar la actividad de gangliosidasas
LT2995306T (lt) 2009-10-19 2019-04-10 Amicus Therapeutics, Inc. Naujos kompozicijos lizosomų kaupimosi sutrikimų profilaktikai ir (arba) gydymui
DK2490532T3 (en) 2009-10-19 2017-02-27 Amicus Therapeutics Inc Hitherto UNKNOWN COMPOSITIONS FOR PREVENTION AND / OR TREATMENT OF DEGENERATIVE DISORDERS IN THE CENTRAL Nervous System
CA2778669C (en) * 2009-10-27 2019-04-16 Erytech Pharma Composition to induce specific immune tolerance
ES2768995T3 (es) * 2009-11-17 2020-06-24 Baylor Res Institute 1-desoxigalactonojirimicina para el uso en el tratamiento de una enfermedad cardíaca
NZ625712A (en) * 2009-11-27 2016-02-26 Genzyme Corp An amorphous and a crystalline form of genz 112638 hemitartrate as inhibitor of glucosylceramide synthase
CN105296447A (zh) 2010-11-08 2016-02-03 阿米库斯治疗学公司 具有增强的稳定性和增强的保留催化活性的变体重组β-葡萄糖脑苷脂酶蛋白
PT2646044T (pt) 2010-11-30 2019-11-12 Orphazyme As Métodos para aumentar a atividade intracelular de hsp70
SG10201604757RA (en) * 2011-03-11 2016-08-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
EP2533051A1 (en) * 2011-06-06 2012-12-12 Centogene GmbH Method for the diagnosis of Gaucher's disease
EP2823043A4 (en) 2012-03-07 2016-09-07 Amicus Therapeutics Inc HIGHLY CONCENTRATED ALPHA-GLUCOSIDASE COMPOSITIONS FOR THE TREATMENT OF MORBUS POMPE
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
EP2874648A4 (en) * 2012-07-17 2015-12-30 Amicus Therapeutics Inc CO-FORMULATION OF ALPHA-GALACTOSIDASE A AND 1-DEOXYGALACTONOJIRIMYCIN
US9675627B2 (en) 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
KR20250069686A (ko) 2014-09-15 2025-05-19 제브라 덴마크 에이/에스 아리모클로몰 제제
BR112017005810A2 (pt) 2014-09-30 2018-02-27 Amicus Therapeutics Inc alfa-glucosidase ácida altamente potente com carboidratos melhorados
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
IL299470A (en) 2015-12-30 2023-02-01 Amicus Therapeutics Inc Improved 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
PE20190127A1 (es) 2016-03-30 2019-01-17 Amicus Therapeutics Inc Metodo para seleccionar proteinas recombinantes ricas en m6p
AU2017239641A1 (en) 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
WO2017178029A1 (en) 2016-04-13 2017-10-19 Orphazyme Aps Heat shock proteins and cholesterol homeostasis
CA3287846A1 (en) 2016-04-29 2025-11-29 Zevra Denmark A/S Arimoclomol for treating glucocerebrosidase associated disorders
CA3023092A1 (en) 2016-05-03 2017-11-09 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.
WO2018200618A1 (en) 2017-04-25 2018-11-01 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating degenerative disorders of the central nervous system and/or lysosomal storage disorders
LT3624831T (lt) 2017-05-15 2023-07-10 Amicus Therapeutics, Inc. Rekombinantinė žmogaus rūgštinė alfa-glukozidazė
KR20240017110A (ko) * 2017-05-30 2024-02-06 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
HUE065615T2 (hu) * 2017-05-30 2024-06-28 Amicus Therapeutics Inc Migalasztát, vesekárosodással szövõdött Fabry-kórban szenvedõ betegeg kezelésére
EP3749307B1 (en) 2018-02-06 2025-12-10 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
CN120044247A (zh) * 2018-12-10 2025-05-27 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
AU2020291002A1 (en) * 2019-06-11 2022-01-06 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having renal impairment
MX2022001623A (es) 2019-08-07 2022-03-02 Amicus Therapeutics Inc Metodos para tratar la enfermedad de fabry en pacientes que tienen una mutacion en el gen gla.
AU2021380947C1 (en) 2020-11-19 2025-02-20 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

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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
EP2444102B1 (en) * 2003-01-31 2015-04-08 The 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
JP2008545657A (ja) * 2005-05-17 2008-12-18 アミカス セラピューティックス インコーポレイテッド １−デオキシノジリマイシンおよび誘導体を用いるポンペ病の治療方法
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
EP2374451A3 (en) * 2005-07-27 2012-01-25 University of Florida Research Foundation, Inc. Histone deacetylase inhibitors (HDAC) that correct protein misfolding and uses thereof
PL2533050T6 (pl) * 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
EP2142197A4 (en) * 2007-03-30 2010-11-10 Amicus Therapeutics Inc PROCESS FOR THE TREATMENT OF FABRY DISEASE USING PHARMACOLOGICAL CHAPERONS

Also Published As

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

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Legal Events

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FA Abandonment or withdrawal