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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JP2016039171A
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English (en)
Japanese (ja)
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JP6672013B2 (ja
JP2016163571A (ja
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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JP2023076515A Withdrawn JP2023109807A (ja) 2008-02-12 2023-05-08 疾病の薬理シャペロン治療に対する応答性を予測する方法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) EP2252313B1 (enExample)
JP (7) JP5844045B2 (enExample)
AU (7) AU2009214648B2 (enExample)
CA (1) CA2715407C (enExample)
CY (3) CY1116466T1 (enExample)
DK (3) DK2252313T3 (enExample)
ES (3) ES2836121T3 (enExample)
HR (3) HRP20150728T1 (enExample)
HU (3) HUE042882T2 (enExample)
LT (2) LT2946785T (enExample)
MX (1) MX2010008835A (enExample)
PL (3) PL2946785T3 (enExample)
PT (3) PT2946785T (enExample)
SI (3) SI2946785T1 (enExample)
WO (1) WO2009102895A2 (enExample)

Families Citing this family (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX351004B (es) 2006-05-16 2017-09-26 Nat Institutes Of Health Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry.
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
AU2009223125A1 (en) * 2008-03-12 2009-09-17 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for Pompe disease
AR078498A1 (es) * 2009-10-01 2011-11-09 Baylor Res Inst Tratamiento de calvicie de patron masculino por induccion local del defecto metabolico de la enfermedad de fabry
US20120283290A1 (en) * 2011-05-06 2012-11-08 Amicus Therapeutics Inc. Quantitation of gl3 in urine
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
EP3270894B1 (en) * 2015-03-19 2021-02-24 Translate Bio, Inc. Mrna therapy for pompe disease
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
ES3063549T3 (en) 2015-12-30 2026-04-17 Amicus Therapeutics Inc Augmented acid alpha-glucosidase for the treatment of pompe disease
IL299470B2 (en) 2015-12-30 2026-05-01 Amicus Therapeutics Inc Improved acid alpha-glucosidase for the treatment of Pompe disease
EP3432882B1 (en) 2016-03-22 2021-09-01 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having the g9331a mutation in the gla gene
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
PL3436053T3 (pl) 2016-03-30 2022-08-01 Amicus Therapeutics, Inc. Formulacje zawierające rekombinowaną kwaśną alfa-glukozydazę
KR102618519B1 (ko) 2016-03-30 2023-12-28 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
WO2018017721A1 (en) 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naïve 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
KR102712926B1 (ko) * 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
EA039750B1 (ru) * 2017-03-30 2022-03-10 Амикус Терапьютикс, Инк. Способ отбора рекомбинантных белков с высоким содержанием m6p
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
AU2018277756A1 (en) 2017-05-30 2020-01-23 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
PL4062916T3 (pl) 2017-05-30 2025-03-17 Amicus Therapeutics, Inc. Leczenie pacjentów z chorobą Fabry’ego mających pewne mutacje
EA202090242A1 (ru) 2017-07-19 2020-05-26 Амикус Терапьютикс, Инк. Лечение болезни фабри у фзт-наивных и фзт-леченных пациентов
WO2019046244A1 (en) 2017-08-28 2019-03-07 Amicus Therapeutics, Inc. METHODS FOR ENHANCING AND / OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE
EP4717319A3 (en) 2018-02-06 2026-06-10 Amicus Therapeutics, Inc. Methods of treating fabry disease in pregnant patients
AU2019217603A1 (en) 2018-02-06 2020-09-24 Amicus Therapeutics, Inc. Treatment of patients with classic Fabry disease
WO2020023390A1 (en) 2018-07-25 2020-01-30 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
LT3840753T (lt) 2018-08-20 2024-11-11 Amicus Therapeutics, Inc. Migalastatas skirtas naudoti fabry ligos gydymo būdui, pacientams turintiems gla geno mutaciją
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
CN113423419A (zh) 2019-01-04 2021-09-21 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
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
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
BR112022004000A2 (pt) 2019-09-06 2022-05-31 Amicus Therapeutics Inc Método para captura e purificação de biológicas
HRP20260399T1 (hr) 2020-02-10 2026-05-08 Amicus Therapeutics, Inc. Postupci liječenja fabryjeve bolesti
FI4114390T3 (fi) * 2020-03-06 2025-03-07 Amicus Therapeutics Inc Menetelmiä fabryn taudin hoitamiseksi potilaissa, joilla on mutaatio gla-geenissä
JP2023531508A (ja) 2020-06-23 2023-07-24 ヴィルテック バイオ,インク. 出血性ショックを処置するための組成物および方法
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
JP2024525760A (ja) 2021-07-12 2024-07-12 アミカス セラピューティックス インコーポレイテッド 小児患者のファブリー病を治療する方法
WO2023215865A1 (en) 2022-05-05 2023-11-09 Amicus Therapeutics, Inc. Methods for treating pompe disease
AR131256A1 (es) 2022-12-02 2025-03-05 Amicus Therapeutics Inc Métodos para tratar la enfermedad de pompe de inicio tardío en pacientes pediátricos
WO2024119091A1 (en) 2022-12-02 2024-06-06 Amicus Therapeutics, Inc. Fexamethods for treating infantile-onset pompe disease in pediatric patients
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
US264467A (en) * 1882-09-19 Chables h
US270486A (en) * 1883-01-09 Bundle-separating attachment for self-binding harvesters
US203019A (en) * 1878-04-30 Improvement in circuits for acoustic or telephonic telegraphs
US21381A (en) * 1858-08-31 Bracelet
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
US6270954B1 (en) 1996-04-10 2001-08-07 The Regents Of The University Of California Correction of genetic defects using chemical chaperones
CA2250780A1 (en) 1996-04-10 1997-10-16 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
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
PL2027137T3 (pl) 2005-06-08 2015-08-31 Amicus Therapeutics Inc Oczyszczanie imino- i aminocukrów
EP1982189A4 (en) * 2006-01-27 2010-09-08 Univ Leland Stanford Junior COMPOSITIONS AND METHODS FOR SCREENING WITH HIGH PASSAGE OF PHARMACOLOGICAL CHAPTERS
MX351004B (es) 2006-05-16 2017-09-26 Nat Institutes Of Health Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry.
WO2007140212A2 (en) 2006-05-24 2007-12-06 Amicus Therapeutics, Inc. Tartrate salt of isofagomine and methods of use
AU2008245578A1 (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
US8321148B2 (en) 2008-10-24 2012-11-27 Amicus Therapeutics, Inc. Multiple compartment dosing model
EP2435459B1 (en) 2009-05-26 2016-05-04 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of acid beta-glucosidase
US9206457B2 (en) 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
EP2502068B1 (en) 2009-11-17 2016-02-10 Baylor Research Institute Urinary triaosylceramide (gb3) as a marker of cardiac disease
WO2012125402A2 (en) 2011-03-11 2012-09-20 Amicus Therapeutics, Inc. Dosing regimens for the treatment of fabry disease
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
EP3432882B1 (en) 2016-03-22 2021-09-01 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having the g9331a mutation in the gla gene
WO2018017721A1 (en) 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naïve and ert-experienced patients
US11357834B2 (en) 2017-01-10 2022-06-14 Amicus Therapeutics, Inc. Recombinant α-galactosidase A for treatment of Fabry disease
AU2018277756A1 (en) 2017-05-30 2020-01-23 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
EA202090242A1 (ru) 2017-07-19 2020-05-26 Амикус Терапьютикс, Инк. Лечение болезни фабри у фзт-наивных и фзт-леченных пациентов
WO2019046244A1 (en) 2017-08-28 2019-03-07 Amicus Therapeutics, Inc. METHODS FOR ENHANCING AND / OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE
AU2019217603A1 (en) 2018-02-06 2020-09-24 Amicus Therapeutics, Inc. Treatment of patients with classic Fabry disease
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
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

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