JP2010525084A5 - - Google Patents

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Publication number
JP2010525084A5
JP2010525084A5 JP2010506557A JP2010506557A JP2010525084A5 JP 2010525084 A5 JP2010525084 A5 JP 2010525084A5 JP 2010506557 A JP2010506557 A JP 2010506557A JP 2010506557 A JP2010506557 A JP 2010506557A JP 2010525084 A5 JP2010525084 A5 JP 2010525084A5
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JP
Japan
Prior art keywords
disease
pharmacological chaperone
use according
days
maintenance dose
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2010506557A
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English (en)
Japanese (ja)
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JP2010525084A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2008/061764 external-priority patent/WO2008134628A2/en
Publication of JP2010525084A publication Critical patent/JP2010525084A/ja
Publication of JP2010525084A5 publication Critical patent/JP2010525084A5/ja
Pending legal-status Critical Current

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JP2010506557A 2007-04-26 2008-04-28 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画 Pending JP2010525084A (ja)

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 (2)

Publication Number Publication Date
JP2010525084A JP2010525084A (ja) 2010-07-22
JP2010525084A5 true JP2010525084A5 (https=) 2011-05-19

Family

ID=39926311

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2010506557A Pending JP2010525084A (ja) 2007-04-26 2008-04-28 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画

Country Status (8)

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

Families Citing this family (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT2441467E (pt) 2003-01-31 2015-10-12 Sinai School Medicine Terapia combinada para o tratamento de transtornos associados a uma deficiência proteica
ES2573498T3 (es) 2006-05-16 2016-06-08 Amicus Therapeutics, Inc. Opciones de tratamiento para la enfermedad de Fabry
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
SI3470077T1 (sl) 2008-02-12 2020-12-31 Amicus Therapeutics, Inc. Postopek za napovedovanje odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
AU2009262670B2 (en) 2008-06-26 2013-06-20 Zevra Denmark A/S 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
US8940766B2 (en) 2009-04-09 2015-01-27 Amicus Therapeutics, Inc. Methods for preventing and/or treating lysosomal storage disorders
GB0906159D0 (en) * 2009-04-09 2009-05-20 Summit Corp Plc Drug combination for the treatment of proteostatic diseases
WO2011049737A1 (en) 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
WO2011049787A1 (en) * 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases
SI2490532T1 (sl) 2009-10-19 2017-03-31 Amicus Therapeutics, Inc. Novi sestavki za preventivo in/ali zdravljenje degenerativnih motenj centralnega živčnega sistema
HRP20161223T1 (hr) * 2009-10-27 2016-11-18 Erytech Pharma Pripravak za induciranje specifične imune tolerancije
JP5739898B2 (ja) * 2009-11-17 2015-06-24 ベイラー リサーチ インスティテュートBaylor Research Institute 心疾患マーカーとしての尿中トリアオシルセラミド(gb3)
WO2011066352A1 (en) 2009-11-27 2011-06-03 Genzyme Corporation An amorphous and a crystalline form of genz 112638 hemitartrat as inhibitor of glucosylceramide synthase
SI2638152T1 (sl) * 2010-11-08 2016-12-30 Amicus Therapeutics, Inc. Variantni in rekombinantni proteini beta-glukocerebrozidaze s povišano stabilnostjo in povišano ohranjeno katalitično aktivnostjo
CA2817773A1 (en) 2010-11-30 2012-06-07 Orphazyme Aps Methods for increasing intracellular activity of hsp70
CN103974619B (zh) * 2011-03-11 2017-02-15 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
KR20200032244A (ko) 2012-03-07 2020-03-25 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
HRP20210398T1 (hr) * 2012-05-03 2021-05-28 Amicus Therapeutics, Inc. Režimi doziranja za liječenje pompeove bolesti
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
AU2015317447B2 (en) 2014-09-15 2021-02-25 Zevra Denmark A/S Arimoclomol formulation
WO2016054231A1 (en) 2014-09-30 2016-04-07 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
EP3344575B1 (en) 2015-09-04 2020-04-15 SQZ Biotechnologies Company Intracellular delivery of biomolecules to cells comprising a cell wall
KR102510941B1 (ko) 2015-12-30 2023-03-20 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
US10227577B2 (en) 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
IL325155A (en) 2016-03-30 2026-02-01 Amicus Therapeutics Inc Formulations containing recombinant acid alpha-glucosidase
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
DK3782624T3 (da) 2016-04-29 2025-12-15 Zevra Denmark As Arimoclomol til behandling af glucocerebrosidase-associerede lidelser
JP7602702B2 (ja) 2016-05-03 2024-12-19 ステムセル テクノロジーズ カナダ インコーポレーテッド 寛容性を誘導するための生体分子の細胞内送達
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
EP3624831B1 (en) 2017-05-15 2023-03-29 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
CA3224537C (en) * 2017-05-30 2025-09-09 Amicus Therapeutics, Inc. USE OF MIGALASTAT IN THE TREATMENT OF PATIENTS WITH FABRY DISEASE AND RENAL FAILURE
PT4324522T (pt) 2017-05-30 2026-03-13 Amicus Therapeutics Inc Métodos de tratamento de pacientes com fabry tendo insuficiência renal
CA3090496C (en) 2018-02-06 2024-03-26 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
EP4282472A3 (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
AR120055A1 (es) * 2019-06-11 2022-02-02 Amicus Therapeutics Inc Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal
EP3851105B1 (en) 2019-08-07 2026-03-04 Amicus Therapeutics, Inc. Migalastat for use in treating fabry disease in patients having a mutation in the gla gene
KR20230128462A (ko) 2020-11-19 2023-09-05 제브라 덴마크 에이/에스 아리모클로몰 시트레이트 및 이의 중간체를 제조하기 위한 공정
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
PT2441467E (pt) * 2003-01-31 2015-10-12 Sinai School Medicine Terapia combinada para o tratamento de transtornos associados a uma 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
EP2932982B1 (en) * 2005-05-17 2018-10-03 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
CN101541172A (zh) 2005-06-08 2009-09-23 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
AU2006272497B2 (en) * 2005-07-27 2012-07-19 University Of Florida Research Foundation, Inc. Small compounds that correct protein misfolding and uses thereof
ES2573498T3 (es) * 2006-05-16 2016-06-08 Amicus Therapeutics, Inc. Opciones de tratamiento para la enfermedad de Fabry
AU2008232614A1 (en) * 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of Fabry disease using pharmacological chaperones

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