JP2016163571A5 - - Google Patents

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Publication number
JP2016163571A5
JP2016163571A5 JP2016039171A JP2016039171A JP2016163571A5 JP 2016163571 A5 JP2016163571 A5 JP 2016163571A5 JP 2016039171 A JP2016039171 A JP 2016039171A JP 2016039171 A JP2016039171 A JP 2016039171A JP 2016163571 A5 JP2016163571 A5 JP 2016163571A5
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JP
Japan
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pharmaceutical composition
galactosidase
specific pharmacological
pharmacological chaperone
gala
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Application number
JP2016039171A
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English (en)
Japanese (ja)
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JP2016163571A (ja
JP6672013B2 (ja
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Publication of JP2016163571A publication Critical patent/JP2016163571A/ja
Publication of JP2016163571A5 publication Critical patent/JP2016163571A5/ja
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JP2016039171A 2008-02-12 2016-03-01 疾病の薬理シャペロン治療に対する応答性を予測する方法 Active JP6672013B2 (ja)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US2814108P 2008-02-12 2008-02-12
US61/028,141 2008-02-12
US3568408P 2008-03-11 2008-03-11
US61/035,684 2008-03-11
US9363108P 2008-09-02 2008-09-02
US61/093,631 2008-09-02
US11349608P 2008-11-11 2008-11-11
US61/113,496 2008-11-11

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2014244283A Division JP2015091239A (ja) 2008-02-12 2014-12-02 疾病の薬理シャペロン治療に対する応答性を予測する方法

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2018245609A Division JP6837469B2 (ja) 2008-02-12 2018-12-27 疾病の薬理シャペロン治療に対する応答性を予測する方法

Publications (3)

Publication Number Publication Date
JP2016163571A JP2016163571A (ja) 2016-09-08
JP2016163571A5 true JP2016163571A5 (enExample) 2016-12-01
JP6672013B2 JP6672013B2 (ja) 2020-03-25

Family

ID=40957500

Family Applications (7)

Application Number Title Priority Date Filing Date
JP2010546901A Active JP5844045B2 (ja) 2008-02-12 2009-02-12 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2014244283A Pending JP2015091239A (ja) 2008-02-12 2014-12-02 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2016039171A Active JP6672013B2 (ja) 2008-02-12 2016-03-01 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2018245609A Active JP6837469B2 (ja) 2008-02-12 2018-12-27 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2021019383A Active JP7277493B2 (ja) 2008-02-12 2021-02-09 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2023076515A Withdrawn JP2023109807A (ja) 2008-02-12 2023-05-08 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2025097940A Pending JP2025148351A (ja) 2008-02-12 2025-06-11 疾病の薬理シャペロン治療に対する応答性を予測する方法

Family Applications Before (2)

Application Number Title Priority Date Filing Date
JP2010546901A Active JP5844045B2 (ja) 2008-02-12 2009-02-12 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2014244283A Pending JP2015091239A (ja) 2008-02-12 2014-12-02 疾病の薬理シャペロン治療に対する応答性を予測する方法

Family Applications After (4)

Application Number Title Priority Date Filing Date
JP2018245609A Active JP6837469B2 (ja) 2008-02-12 2018-12-27 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2021019383A Active JP7277493B2 (ja) 2008-02-12 2021-02-09 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2023076515A Withdrawn JP2023109807A (ja) 2008-02-12 2023-05-08 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2025097940A Pending JP2025148351A (ja) 2008-02-12 2025-06-11 疾病の薬理シャペロン治療に対する応答性を予測する方法

Country Status (16)

Country Link
US (8) US8592362B2 (enExample)
EP (4) EP2946785B1 (enExample)
JP (7) JP5844045B2 (enExample)
AU (7) AU2009214648B2 (enExample)
CA (1) CA2715407C (enExample)
CY (3) CY1116466T1 (enExample)
DK (3) DK2946785T3 (enExample)
ES (3) ES2716502T3 (enExample)
HR (3) HRP20150728T1 (enExample)
HU (3) HUE026543T2 (enExample)
LT (2) LT2946785T (enExample)
MX (1) MX2010008835A (enExample)
PL (3) PL2252313T3 (enExample)
PT (3) PT3470077T (enExample)
SI (3) SI3470077T1 (enExample)
WO (1) WO2009102895A2 (enExample)

Families Citing this family (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
WO2009114712A2 (en) * 2008-03-12 2009-09-17 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for pompe disease
TW201117810A (en) * 2009-10-01 2011-06-01 Baylor Res Inst Treatment of male-pattern baldness by local induction of the metabolic defect of fabry disease
US20120283290A1 (en) * 2011-05-06 2012-11-08 Amicus Therapeutics Inc. Quantitation of gl3 in urine
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
WO2016149508A1 (en) 2015-03-19 2016-09-22 Shire Human Genetic Therapies, Inc. Mrna therapy for pompe disease
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
RS64843B1 (sr) 2015-12-30 2023-12-29 Amicus Therapeutics Inc Augmentirana kisela alfa-glukozidaza za lečenje pompeove bolesti
KR102747683B1 (ko) 2015-12-30 2024-12-31 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
KR20190087673A (ko) * 2016-03-22 2019-07-24 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 g9331a 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
SG11201808455VA (en) 2016-03-30 2018-10-30 Amicus Therapeutics Inc Formulations comprising recombinant acid alpha-glucosidase
DK3436053T3 (da) 2016-03-30 2022-07-11 Amicus Therapeutics Inc Formuleringer, som omfatter rekombinant syre-alfa-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
AU2017298376A1 (en) 2016-07-19 2019-03-07 Amicus Therapeutics, Inc. Treatment of Fabry disease in ERT-naive and ERT-experienced patients
JP6546135B2 (ja) 2016-08-24 2019-07-17 株式会社ノリタケカンパニーリミテド 積層造形焼成体、積層造形焼成体の製造方法および積層造形焼成体製造用キット
CN110022904B (zh) * 2016-10-20 2024-04-19 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
EA039750B1 (ru) * 2017-03-30 2022-03-10 Амикус Терапьютикс, Инк. Способ отбора рекомбинантных белков с высоким содержанием m6p
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
WO2019017938A1 (en) 2017-07-19 2019-01-24 Amicus Therapeutics, Inc. TREATMENT OF FABRY'S DISEASE IN PATIENTS WITHOUT SUBSTITUTED ENZYMATIC THERAPY (TES) AND PATIENTS SUBJECT TO TES
PT3675853T (pt) 2017-08-28 2022-11-28 Amicus Therapeutics Inc Métodos para melhorar e/ou estabilizar a função cardíaca em pacientes com doença de fabry
PT3749308T (pt) 2018-02-06 2024-01-25 Amicus Therapeutics Inc Tratamento de pacientes com doença de fabry clássica com migalastato
EP3749307B1 (en) 2018-02-06 2025-12-10 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
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
NL2021840B1 (en) 2018-10-19 2020-05-13 Univ Leiden Pharmacological Chaperones For Enzyme Treatment Therapy
WO2020046132A1 (en) 2018-08-31 2020-03-05 Leiden University Pharmacological chaperones for enzyme treatment therapy
TW202042812A (zh) 2019-01-22 2020-12-01 美商阿米庫斯醫療股份有限公司 減少法布瑞氏症患者中之腦血管事件之方法
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
KR20220058587A (ko) 2019-09-06 2022-05-09 아미쿠스 세라퓨틱스, 인코포레이티드 생물학적 제제의 포획 및 정제 방법
PL4103178T3 (pl) 2020-02-10 2026-05-11 Amicus Therapeutics, Inc. Sposoby leczenia choroby fabry'ego
SI4114390T1 (sl) * 2020-03-06 2025-03-31 Amicus Therapeutics, Inc. Postopki zdravljenja Fabryjeve bolezni pri bolnikih z mutacijo gena GLA
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
WO2023288210A1 (en) 2021-07-12 2023-01-19 Amicus Therapeutics, Inc. Methods of treating fabry disease in pediatric patients
AU2023265155A1 (en) 2022-05-05 2024-12-12 Amicus Therapeutics, Inc. Methods for treating pompe disease
EP4626464A1 (en) 2022-12-02 2025-10-08 Amicus Therapeutics, Inc. Fexamethods for treating infantile-onset pompe disease in pediatric patients
KR20250113510A (ko) 2022-12-02 2025-07-25 아미쿠스 세라퓨틱스, 인코포레이티드 소아 환자의 후기 발병 폼페병 치료에 대한 방법
US20240197706A1 (en) 2022-12-13 2024-06-20 Amicus Therapeutics, Inc. Methods of improving the pharmacokinetics of migalastat
WO2026006625A1 (en) 2024-06-26 2026-01-02 Amicus Therapeutics, Inc. Miglustat formulations comprising nitrosamine for use in the treatment of pompe disease

Family Cites Families (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US203019A (en) * 1878-04-30 Improvement in circuits for acoustic or telephonic telegraphs
US270486A (en) * 1883-01-09 Bundle-separating attachment for self-binding harvesters
US21381A (en) * 1858-08-31 Bracelet
US264467A (en) * 1882-09-19 Chables h
US3586908A (en) 1969-02-28 1971-06-22 Robert E Vosteen Automatic potential control system for electrophotography apparatus
US5401650A (en) * 1990-10-24 1995-03-28 The Mount Sinai School Of Medicine Of The City University Of New York Cloning and expression of biologically active α-galactosidase A
US5863903A (en) 1994-03-09 1999-01-26 Novo Nordisk A/S Use of hydroxy alkyl piperidine and pyrrolidine compounds to treat diabetes
US5844102A (en) 1994-07-07 1998-12-01 University Of Maryland Baltimore County Glycohydrolase inhibitors, their preparation and use thereof
US5900360A (en) 1996-04-10 1999-05-04 Welch; William J. Correction of genetic defects using chemical chaperones
US6270954B1 (en) 1996-04-10 2001-08-07 The Regents Of The University Of California Correction of genetic defects using chemical chaperones
US5948653A (en) 1997-03-21 1999-09-07 Pati; Sushma Sequence alterations using homologous recombination
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
US6244113B1 (en) 1999-10-29 2001-06-12 University Of Alabama In Huntsville Method and apparatus for measuring microgravity acceleration
US7842470B2 (en) * 2001-10-09 2010-11-30 Oregon Health & Science University Method for pharmacoperones correction of GnRHR mutant protein misfolding
AU2006247065B8 (en) * 2005-05-17 2012-07-12 Amicus Therapeutics, Inc. A method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives
US7973157B2 (en) 2005-06-08 2011-07-05 Amicus Therapeutics, Inc. Imino and amino sugar purification
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.
WO2007089591A2 (en) * 2006-01-27 2007-08-09 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for high throughput screening of pharmacological chaperones
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
US7501439B2 (en) 2006-05-24 2009-03-10 Amicus Therapeutics, Inc. Tartrate salt of isofagomine and methods of use
WO2008134628A2 (en) 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
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
WO2010048532A1 (en) 2008-10-24 2010-04-29 Amicus Therapeutics, Inc. Multiple compartment dosing model
WO2010138608A1 (en) 2009-05-26 2010-12-02 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
US9206457B2 (en) 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
AR079057A1 (es) 2009-11-17 2011-12-21 Baylor Res Inst Triaosilceramida urinaria (gb3) como marcador de enfermedad cardiaca
US20140219986A1 (en) 2011-03-11 2014-08-07 Amicus Therapeutics ,Inc. Dosing regimens for the treatment of fabry 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
KR20190087673A (ko) 2016-03-22 2019-07-24 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 g9331a 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
AU2017298376A1 (en) 2016-07-19 2019-03-07 Amicus Therapeutics, Inc. Treatment of Fabry disease in ERT-naive and ERT-experienced patients
TW201829770A (zh) 2017-01-10 2018-08-16 美商阿米庫斯醫療股份有限公司 用於治療fabry氏病之重組α-半乳糖苷酶A
KR20220116305A (ko) 2017-05-30 2022-08-22 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
WO2019017938A1 (en) 2017-07-19 2019-01-24 Amicus Therapeutics, Inc. TREATMENT OF FABRY'S DISEASE IN PATIENTS WITHOUT SUBSTITUTED ENZYMATIC THERAPY (TES) AND PATIENTS SUBJECT TO TES
PT3675853T (pt) 2017-08-28 2022-11-28 Amicus Therapeutics Inc Métodos para melhorar e/ou estabilizar a função cardíaca em pacientes com doença de fabry
EP3749307B1 (en) 2018-02-06 2025-12-10 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
PT3749308T (pt) 2018-02-06 2024-01-25 Amicus Therapeutics Inc Tratamento de pacientes com doença de fabry clássica com migalastato
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
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

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