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
Other languages
English (en)
Japanese (ja)
Other versions
JP2010525084A (ja
Filing date
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

Links

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 (enExample) 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 (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)

Families Citing this family (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5589165B2 (ja) 2003-01-31 2014-09-17 マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー タンパク質欠損性障害の治療のための併用療法
MX351004B (es) 2006-05-16 2017-09-26 Nat Institutes Of Health Ensayos para diagnosticar y evaluar opciones de tratamiento para 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
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
JP5616336B2 (ja) 2008-06-26 2014-10-29 オルファザイム エーピーエスOrphazyme Aps 酵素活性の調節因子としてのHsp70の使用
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
JP5645918B2 (ja) 2009-04-09 2014-12-24 アミカス セラピューティックス インコーポレイテッド リソソーム蓄積障害の予防および/または治療方法
DK2490712T3 (en) * 2009-10-19 2015-10-05 Amicus Therapeutics Inc PROCESS FOR THE TREATMENT OF ALZHEIMER'S DISEASE BY USING Pharmacological chaperones TO INCREASE ACTIVITY OF GANGLIOSIDASER
WO2011049737A1 (en) 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
RU2015147509A (ru) 2009-10-19 2019-01-14 Амикус Терапьютикс, Инк. Новые композиции для предупреждения и/или лечения дегенеративных расстройств центральной нервной системы
ES2600932T3 (es) * 2009-10-27 2017-02-13 Erytech Pharma Composición para inducir una tolerancia inmunitaria específica
EP2502068B1 (en) * 2009-11-17 2016-02-10 Baylor Research Institute Urinary triaosylceramide (gb3) as a marker of cardiac disease
MY192644A (en) * 2009-11-27 2022-08-29 Genzyme Corp An amorphous and a crystalline form of genz 112638 hemitartrate as inhibitor of glucosylceramide synthase
US8962564B2 (en) 2010-11-08 2015-02-24 Amicus Therapeutics, Inc. Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity
US9662375B2 (en) 2010-11-30 2017-05-30 Orphazyme Aps Methods for increasing intracellular activity of Hsp70
WO2012125402A2 (en) * 2011-03-11 2012-09-20 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
KR20200032244A (ko) 2012-03-07 2020-03-25 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.
RS61598B1 (sr) * 2012-05-03 2021-04-29 Amicus Therapeutics Inc Režimi doziranja za tretman pompeove bolesti
EP2874648A4 (en) 2012-07-17 2015-12-30 Amicus Therapeutics Inc ALPHA GALACTOSIDASE A AND 1-DEOXYGALACTONO JIRIMYCIN 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
JP6851964B2 (ja) 2014-09-30 2021-03-31 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ
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
US11613759B2 (en) 2015-09-04 2023-03-28 Sqz Biotechnologies Company Intracellular delivery of biomolecules to cells comprising a cell wall
IL299470B2 (en) 2015-12-30 2026-05-01 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
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
KR102618519B1 (ko) 2016-03-30 2023-12-28 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
WO2017178029A1 (en) 2016-04-13 2017-10-19 Orphazyme Aps Heat shock proteins and cholesterol homeostasis
ES2831764T3 (es) 2016-04-29 2021-06-09 Orphazyme As Arimoclomol para el tratamiento de trastornos asociados a glucocerebrosidasa
CN109415741A (zh) 2016-05-03 2019-03-01 Sqz生物技术公司 细胞内递送生物分子以诱导耐受性
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
CN119113091A (zh) 2017-05-15 2024-12-13 阿米库斯治疗学公司 重组人类酸性α-葡萄糖苷酶
PL4062916T3 (pl) 2017-05-30 2025-03-17 Amicus Therapeutics, Inc. Leczenie pacjentów z chorobą Fabry’ego mających pewne mutacje
AU2018277756A1 (en) 2017-05-30 2020-01-23 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
EP4717319A3 (en) 2018-02-06 2026-06-10 Amicus Therapeutics, Inc. Methods of treating fabry disease in pregnant patients
LT3840753T (lt) 2018-08-20 2024-11-11 Amicus Therapeutics, Inc. Migalastatas skirtas naudoti fabry ligos gydymo būdui, pacientams turintiems gla geno mutaciją
EP3894864A2 (en) * 2018-12-10 2021-10-20 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
KR20220044560A (ko) 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
EP4247792A1 (en) 2020-11-19 2023-09-27 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
US20240197706A1 (en) 2022-12-13 2024-06-20 Amicus Therapeutics, Inc. Methods of improving the pharmacokinetics of migalastat

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
JP5589165B2 (ja) * 2003-01-31 2014-09-17 マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー タンパク質欠損性障害の治療のための併用療法
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
EP3782655A1 (en) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
ES2485369T3 (es) * 2005-06-08 2014-08-13 Amicus Therapeutics, Inc. Tratamiento de trastornos del SNC asociados con mutaciones en genes que codifican enzimas lisosómicas
US7955262B2 (en) 2005-07-26 2011-06-07 Syneron Medical Ltd. Method and apparatus for treatment of skin using RF and ultrasound energies
CN101267779A (zh) * 2005-07-27 2008-09-17 福罗里达大学研究基金会有限公司 使用热休克治疗眼睛疾病
MX351004B (es) * 2006-05-16 2017-09-26 Nat Institutes Of Health Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry.
EP2142197A4 (en) 2007-03-30 2010-11-10 Amicus Therapeutics Inc METHOD FOR THE TREATMENT OF MORBUS FABRY WITH PHARMACOLOGICAL CHAPTERONES

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