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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Publication date
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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 (enExample) 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 (enExample)
EP (1) EP2416656B1 (enExample)
JP (1) JP5645918B2 (enExample)
CN (1) CN102625661B (enExample)
AU (1) AU2010233188B2 (enExample)
BR (1) BRPI1010517A2 (enExample)
CA (1) CA2758271C (enExample)
ES (1) ES2564093T3 (enExample)
IL (1) IL215583A (enExample)
NZ (1) NZ595629A (enExample)
WO (1) WO2010118283A1 (enExample)

Families Citing this family (12)

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Publication number Priority date Publication date Assignee Title
EP3782655A1 (en) 2005-05-17 2021-02-24 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
RU2015147509A (ru) 2009-10-19 2019-01-14 Амикус Терапьютикс, Инк. Новые композиции для предупреждения и/или лечения дегенеративных расстройств центральной нервной системы
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
CA2939380A1 (en) * 2014-02-12 2015-08-20 Amicus Therapeutics, Inc. Sugar derivatives comprising sulfur-containing moieties and methods of making same and methods of using the same for the treatment of mps iiic
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
IL299470B2 (en) 2015-12-30 2026-05-01 Amicus Therapeutics Inc Improved acid alpha-glucosidase for the treatment of Pompe disease
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
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
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

Family Cites Families (20)

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US4407957A (en) 1981-03-13 1983-10-04 Damon Corporation Reversible microencapsulation of a core material
SG47470A1 (en) 1991-04-25 1998-04-17 Univ Brown Res Found Implantable biocompatible immunoisolatory vehicle for delivery of a 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
DK0679088T3 (da) 1992-09-29 2002-10-28 Inhale Therapeutic Syst Pulmonal levering af aktive fragmenter af parathyreoideaea hormon
AU1890295A (en) * 1994-03-09 1995-09-25 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
WO2002076440A2 (en) * 2001-03-23 2002-10-03 Elan Pharmaceuticals, Inc. Methods of treating alzheimer's disease with piperidin derivates
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
US20050137223A1 (en) * 2003-11-12 2005-06-23 Amicus Therapeutics, Inc. Glucoimidazole and polyhydroxycyclohexenyl amine derivatives to treat gaucher disease
DK2040548T3 (da) * 2006-06-23 2012-08-13 Amicus Therapeutics Inc Fremgangsmåde til behandling af neurologiske forstyrrelser ved forøgelse af aktiviteten af beta-glucocerebrosidase
AU2008245578A1 (en) 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
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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