IL201733A0 - Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones - Google Patents

Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones

Info

Publication number
IL201733A0
IL201733A0 IL201733A IL20173309A IL201733A0 IL 201733 A0 IL201733 A0 IL 201733A0 IL 201733 A IL201733 A IL 201733A IL 20173309 A IL20173309 A IL 20173309A IL 201733 A0 IL201733 A0 IL 201733A0
Authority
IL
Israel
Prior art keywords
treatment
lysosomal storage
dosing regimens
storage diseases
pharmacological chaperones
Prior art date
Application number
IL201733A
Other languages
English (en)
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 IL201733A0 publication Critical patent/IL201733A0/en

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)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Hospice & Palliative Care (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Psychiatry (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)
IL201733A 2007-04-26 2009-10-25 Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones IL201733A0 (en)

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
IL201733A0 true IL201733A0 (en) 2010-06-16

Family

ID=39926311

Family Applications (1)

Application Number Title Priority Date Filing Date
IL201733A IL201733A0 (en) 2007-04-26 2009-10-25 Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones

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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PT2857036T (pt) 2003-01-31 2017-03-13 Mount Sinai School Of Medicine Of N Y Univ Terapia combinada para o tratamento de distúrbios de deficiência proteica
JP2009537149A (ja) 2006-05-16 2009-10-29 アミークス セラピューティックス インコーポレイテッド ファブリー病に対する治療の選択を診断し、評価するためのアッセイ
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
EP2484371B1 (en) 2008-06-26 2014-12-17 Orphazyme APS Use of Hsp70 as a regulator of enzymatic activity
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
BRPI1010517A2 (pt) 2009-04-09 2017-01-31 Amicus Therapeutics Inc métodos para prevenção e/ou tratamento de doenças de armazenamento lisossomal
GB0906159D0 (en) * 2009-04-09 2009-05-20 Summit Corp Plc Drug combination for the treatment of proteostatic diseases
PT2995306T (pt) 2009-10-19 2019-03-29 Amicus Therapeutics Inc Novas composições para prevenção e/ou tratamento de disfunções de armazenamento lisossomal
JP6061677B2 (ja) * 2009-10-19 2017-01-18 アミカス セラピューティックス インコーポレイテッド 薬理シャペロンを用いてガングリオシドの活性を増大させて、アルツハイマー病を治療するための方法
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
WO2011051346A1 (en) * 2009-10-27 2011-05-05 Erytech Pharma Composition to induce specific immune tolerance
AR079057A1 (es) * 2009-11-17 2011-12-21 Baylor Res Inst Triaosilceramida urinaria (gb3) como marcador de enfermedad cardiaca
SG10201407881WA (en) 2009-11-27 2015-01-29 Genzyme Corp An amorphous and a crystalline form of genz 112638 hemitartrate as inhibitor of glucosylceramide synthase
HUE030932T2 (en) 2010-11-08 2017-06-28 Amicus Therapeutics Inc Increased stability and increased catalytic activity, altered, recombinant, β-glucocerebrosidase proteins
RU2013125923A (ru) 2010-11-30 2015-01-10 Орфазиме Апс СПОСОБЫ УВЕЛИЧЕНИЯ ВНУТРИКЛЕТОЧНОЙ АКТИВНОСТИ Hsp70
US20140219986A1 (en) * 2011-03-11 2014-08-07 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
KR20230066482A (ko) 2012-03-07 2023-05-15 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
CA2868459A1 (en) 2012-03-27 2013-10-03 Amicus Therapeutics, Inc. Polymorphs of (3r,4r,5s)-5-(difluoromethyl)piperidine-3,4-diol fumarate and uses thereof
EP2844279B1 (en) * 2012-05-03 2020-12-09 Amicus Therapeutics, Inc. Dosing regimens for the treatment of pompe disease
HK1209625A1 (en) 2012-07-17 2016-04-08 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
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
US9675627B2 (en) * 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
US10709700B2 (en) 2014-09-15 2020-07-14 Orphazyme A/S Arimoclomol formulation
CN114540327B (zh) 2014-09-30 2026-03-24 阿米库斯治疗学公司 具有增强的碳水化合物的高强度酸性α-葡糖苷酶
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
KR102747683B1 (ko) 2015-12-30 2024-12-31 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
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
SG11201808455VA (en) 2016-03-30 2018-10-30 Amicus Therapeutics Inc Formulations comprising recombinant acid alpha-glucosidase
JP7046003B2 (ja) 2016-03-30 2022-04-01 アミカス セラピューティックス インコーポレイテッド 高ｍ６ｐ組換えタンパク質の選択方法
EP3442530A1 (en) 2016-04-13 2019-02-20 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
SI3448382T1 (sl) 2016-04-29 2021-03-31 Orphazyme A/S, c/o COBIS A/S Arimoclomol za zdravljenje motenj, povezanih z glukocerebrozidazo
RU2770492C2 (ru) 2016-05-03 2022-04-18 ЭсКьюЗед БАЙОТЕКНОЛОДЖИЗ КОМПАНИ Внутриклеточная доставка биологических молекул для индуцирования толерантности
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
ES2950808T3 (es) 2017-05-15 2023-10-13 Amicus Therapeutics Inc Alfa-glucosidasa ácida humana recombinante
PT4324522T (pt) * 2017-05-30 2026-03-13 Amicus Therapeutics Inc Métodos de tratamento de pacientes com fabry tendo insuficiência renal
KR20220116305A (ko) 2017-05-30 2022-08-22 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
EP3749307B1 (en) 2018-02-06 2025-12-10 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
EP4289476A3 (en) 2018-08-20 2024-02-21 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
EP3894864A2 (en) * 2018-12-10 2021-10-20 Denali Therapeutics Inc. Lysosomal storage disorder biomarkers and methods of use thereof
BR112021024886A2 (pt) * 2019-06-11 2022-01-25 Amicus Therapeutics Inc Métodos de tratar a doença de fabry em pacientes com insuficiência renal
WO2021026447A1 (en) 2019-08-07 2021-02-11 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
WO2022106614A1 (en) 2020-11-19 2022-05-27 Orphazyme 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
PT2857036T (pt) * 2003-01-31 2017-03-13 Mount Sinai School Of Medicine Of N Y Univ Terapia combinada para o tratamento de distúrbios de deficiência proteica
US7446098B2 (en) * 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
AU2006247065B8 (en) * 2005-05-17 2012-07-12 Amicus Therapeutics, Inc. A method for the treatment of Pompe disease using 1-deoxynojirimycin 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.
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Also Published As

Publication number Publication date
JP2010525084A (ja) 2010-07-22
WO2008134628A2 (en) 2008-11-06
US9056101B2 (en) 2015-06-16
CA2685332A1 (en) 2008-11-06
EP2150254A4 (en) 2010-11-10
US20100266571A1 (en) 2010-10-21
EP2150254A2 (en) 2010-02-10
WO2008134628A3 (en) 2010-01-14
US20150352093A1 (en) 2015-12-10
MX2009011473A (es) 2010-01-18
AU2008245578A1 (en) 2008-11-06

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