JP2012523431A5 - - Google Patents

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Publication number
JP2012523431A5
JP2012523431A5 JP2012504881A JP2012504881A JP2012523431A5 JP 2012523431 A5 JP2012523431 A5 JP 2012523431A5 JP 2012504881 A JP2012504881 A JP 2012504881A JP 2012504881 A JP2012504881 A JP 2012504881A JP 2012523431 A5 JP2012523431 A5 JP 2012523431A5
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JP
Japan
Prior art keywords
lysosomal storage
diol
storage disorder
prodrug
solvate
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Application number
JP2012504881A
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English (en)
Japanese (ja)
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JP5645918B2 (ja
JP2012523431A (ja
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Priority claimed from PCT/US2010/030472 external-priority patent/WO2010118283A1/en
Publication of JP2012523431A publication Critical patent/JP2012523431A/ja
Publication of JP2012523431A5 publication Critical patent/JP2012523431A5/ja
Application granted granted Critical
Publication of JP5645918B2 publication Critical patent/JP5645918B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2012504881A 2009-04-09 2010-04-09 リソソーム蓄積障害の予防および/または治療方法 Expired - Fee Related JP5645918B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US16810909P 2009-04-09 2009-04-09
US61/168,109 2009-04-09
PCT/US2010/030472 WO2010118283A1 (en) 2009-04-09 2010-04-09 Methods for preventing and/or treating lysosomal storage disorders

Publications (3)

Publication Number Publication Date
JP2012523431A JP2012523431A (ja) 2012-10-04
JP2012523431A5 true JP2012523431A5 (https=) 2013-05-16
JP5645918B2 JP5645918B2 (ja) 2014-12-24

Family

ID=42934559

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2012504881A Expired - Fee Related JP5645918B2 (ja) 2009-04-09 2010-04-09 リソソーム蓄積障害の予防および/または治療方法

Country Status (11)

Country Link
US (1) US8940766B2 (https=)
EP (1) EP2416656B1 (https=)
JP (1) JP5645918B2 (https=)
CN (1) CN102625661B (https=)
AU (1) AU2010233188B2 (https=)
BR (1) BRPI1010517A2 (https=)
CA (1) CA2758271C (https=)
ES (1) ES2564093T3 (https=)
IL (1) IL215583A (https=)
NZ (1) NZ595629A (https=)
WO (1) WO2010118283A1 (https=)

Families Citing this family (12)

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EP2932982B1 (en) 2005-05-17 2018-10-03 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
CA2758187C (en) * 2009-04-09 2017-03-07 Robert Boyd Methods for preventing and/or treating degenerative disorders of the central nervous system
SI2490532T1 (sl) 2009-10-19 2017-03-31 Amicus Therapeutics, Inc. Novi sestavki za preventivo in/ali zdravljenje degenerativnih motenj centralnega živčnega sistema
WO2011049737A1 (en) 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
KR20200032244A (ko) * 2012-03-07 2020-03-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
ES2771849T3 (es) * 2014-02-12 2020-07-07 Amicus Therapeutics Inc Derivados de azúcares que comprenden restos que contienen azufre, métodos para prepararlos y métodos para emplearlos en el tratamiento de MPS IIIC
WO2016054231A1 (en) 2014-09-30 2016-04-07 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
KR102510941B1 (ko) 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
IL325155A (en) 2016-03-30 2026-02-01 Amicus Therapeutics Inc Formulations containing recombinant acid alpha-glucosidase
US10227577B2 (en) 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
EP3624831B1 (en) 2017-05-15 2023-03-29 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
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

Family Cites Families (20)

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US4407957A (en) 1981-03-13 1983-10-04 Damon Corporation Reversible microencapsulation of a core material
CA2109085C (en) 1991-04-25 2003-03-11 Keith E. Dionne Implantable biocompatible immunoisolatory vehicle for delivery of selected therapeutic products
US5383851A (en) 1992-07-24 1995-01-24 Bioject Inc. Needleless hypodermic injection device
US5569189A (en) 1992-09-28 1996-10-29 Equidyne Systems, Inc. hypodermic jet injector
ES2179831T3 (es) 1992-09-29 2003-02-01 Inhale Therapeutic Syst Liberacion en los pulmones de fragmentos activos de hormona paratiroidiana.
WO1995024391A1 (en) * 1994-03-09 1995-09-14 Novo Nordisk A/S Piperidines and pyrrolidines
FR2718357B1 (fr) 1994-04-06 1997-10-03 Defarges Alain Moreau Perfectionnements apportés à un dispositif d'injection par jet sans aiguille.
US5844102A (en) 1994-07-07 1998-12-01 University Of Maryland Baltimore County Glycohydrolase inhibitors, their preparation and use thereof
US5599302A (en) 1995-01-09 1997-02-04 Medi-Ject Corporation Medical injection system and method, gas spring thereof and launching device using gas spring
US5780014A (en) 1995-04-14 1998-07-14 Inhale Therapeutic Systems Method and apparatus for pulmonary administration of dry powder alpha 1-antitrypsin
US5654007A (en) 1995-06-07 1997-08-05 Inhale Therapeutic Systems Methods and system for processing dispersible fine powders
US20040204379A1 (en) 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20060079533A1 (en) * 2001-03-23 2006-04-13 Nieman James A Methods of treating alzheimer's disease
JP2006507359A (ja) 2002-10-28 2006-03-02 ノボ・ノルデイスク・エー/エス 心循環器疾患の治療のためのグリコーゲンホスホリラーゼ阻害剤の使用
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
EP1682504B1 (en) * 2003-11-12 2012-12-26 Amicus Therapeutics Inc. Hydroxy piperidine derivatives to treat gaucher disease
JP5303458B2 (ja) * 2006-06-23 2013-10-02 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 β−グルコセレブロシダーゼの活性増強による神経学的疾患の治療方法
EP2150254A4 (en) 2007-04-26 2010-11-10 Amicus Therapeutics Inc DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH PHARMACOLOGICAL CHAPTERONES
US8321148B2 (en) 2008-10-24 2012-11-27 Amicus Therapeutics, Inc. Multiple compartment dosing model
CA2758187C (en) * 2009-04-09 2017-03-07 Robert Boyd Methods for preventing and/or treating degenerative disorders of the central nervous system

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