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)
  • Cardiology (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Obesity (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 (enExample)
EP (1) EP2150254A4 (enExample)
JP (1) JP2010525084A (enExample)
AU (1) AU2008245578A1 (enExample)
CA (1) CA2685332A1 (enExample)
IL (1) IL201733A0 (enExample)
MX (1) MX2009011473A (enExample)
WO (1) WO2008134628A2 (enExample)

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WO2004069190A2 (en) 2003-01-31 2004-08-19 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiency disorders
WO2007137072A2 (en) 2006-05-16 2007-11-29 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
AU2009214648B2 (en) 2008-02-12 2014-11-13 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
CA2728363C (en) 2008-06-26 2019-02-19 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
US20100119502A1 (en) * 2008-11-11 2010-05-13 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
NZ595629A (en) 2009-04-09 2013-04-26 Amicus Therapeutics Inc Methods for preventing and/or treating lysosomal storage disorders
MX337933B (es) 2009-10-19 2016-03-29 Amicus Therapeutics Inc Nuevas composiciones para prevenir y/o tratar trastornos de almacenamiento lisosomal.
KR101769396B1 (ko) 2009-10-19 2017-08-18 아미쿠스 세라퓨틱스, 인코포레이티드 중추신경계의 퇴행성 질환을 예방 및/또는 치료하는 신규 조성물
PL2490712T3 (pl) 2009-10-19 2015-12-31 Amicus Therapeutics Inc Sposób leczenia choroby Alzheimera przy zastosowaniu chaperonów farmakologicznych dla zwiększenia aktywności gangliozydaz
PL2493487T3 (pl) * 2009-10-27 2017-02-28 Erytech Pharma Kompozycja do indukowania swoistej tolerancji immunologicznej
WO2011063048A2 (en) * 2009-11-17 2011-05-26 Baylor Research Institute Urinary triaosylceramide (gb3) as a marker of cardiac disease
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
DK2638152T3 (en) 2010-11-08 2016-12-12 Amicus Therapeutics Inc VARIANTE, RECOMBINANT BETA-glucocerebrosidase PROTEINS WITH INCREASED STABILITY AND INCREASED RETAINED CATALYTIC ACTIVITY
PL2646044T3 (pl) 2010-11-30 2020-03-31 Orphazyme A/S Sposoby zwiększenia aktywności wewnątrzkomórkowej Hsp70
CN107088225A (zh) 2011-03-11 2017-08-25 阿米库斯治疗学公司 治疗法布里病的给药方案
EP2533051A1 (en) * 2011-06-06 2012-12-12 Centogene GmbH Method for the diagnosis of Gaucher's disease
KR20140135222A (ko) 2012-03-07 2014-11-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
MX354127B (es) 2012-03-27 2018-02-14 Amicus Therapeutics Inc Compuestos novedosos para prevenir y/o tratar tesaurismosis lisosomicas y/o trastornos degenerativos del sistema nervioso central.
ES2859761T3 (es) * 2012-05-03 2021-10-04 Amicus Therapeutics Inc Pautas posológicas para el tratamiento de enfermedad de Pompe
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
JP6678676B2 (ja) 2014-09-15 2020-04-08 オーファザイム エー／エス アリモクロモル製剤
DK3201320T5 (da) 2014-09-30 2024-10-14 Amicus Therapeutics Inc Højpotent sur alfa-glucosidase med forbedrede carbohydrater
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
MX394968B (es) 2015-12-30 2025-03-24 Amicus Therapeutics Inc Alfa-glucosidasa con mayor cantidad de acido para el tratamiento de la enfermedad de pompe.
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
KR102343162B1 (ko) 2016-03-30 2021-12-23 아미쿠스 세라퓨틱스, 인코포레이티드 고 m6p 재조합 단백질의 선택 방법
CA3019128A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
BR112018070653A2 (pt) 2016-04-29 2019-02-05 Orphazyme As ingrediente farmacêutico ativo, e, composição
KR102430856B1 (ko) 2016-05-03 2022-08-08 에스큐지 바이오테크놀로지스 컴퍼니 관용을 유도하는 생체분자의 세포내 전달
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
KR20190134807A (ko) 2017-04-25 2019-12-04 아미쿠스 세라퓨틱스, 인코포레이티드 중추 신경계의 퇴행성 질환 및/또는 리소솜 저장병을 예방 및/또는 치료하기 위한 신규한 조성물
SMT202300187T1 (it) 2017-05-15 2023-07-20 Amicus Therapeutics Inc Alfa-glucosidasi acida umana ricombinante
TWI795408B (zh) 2017-05-30 2023-03-11 美商阿米庫斯醫療股份有限公司 治療有腎損傷的法布里患者的方法
CA3224529A1 (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
US20220025065A1 (en) * 2018-12-10 2022-01-27 Denali Therapeutics Inc. Lysosomal storage disorder biomarkers and methods of use thereof
US20220313670A1 (en) * 2019-06-11 2022-10-06 Amicus Therapeutics, Inc. Methods Of Treating Fabry Disease In Patients Having Renal Impairment
US11833164B2 (en) 2019-08-07 2023-12-05 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
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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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
ES2572148T3 (es) * 2005-05-17 2016-05-30 Amicus Therapeutics Inc Un método para el tratamiento de la enfermedad de Pompe usando 1-desoxinojirimicina y derivados
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
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Also Published As

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

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Legal Events

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FA Abandonment or withdrawal