JP2015509961A5 - - Google Patents

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JP2015509961A5
JP2015509961A5 JP2014560086A JP2014560086A JP2015509961A5 JP 2015509961 A5 JP2015509961 A5 JP 2015509961A5 JP 2014560086 A JP2014560086 A JP 2014560086A JP 2014560086 A JP2014560086 A JP 2014560086A JP 2015509961 A5 JP2015509961 A5 JP 2015509961A5
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JP
Japan
Prior art keywords
pharmaceutical composition
subject
replacement therapy
gaucher disease
enzyme replacement
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JP2014560086A
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English (en)
Japanese (ja)
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JP2015509961A (ja
JP6230160B2 (ja
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Priority claimed from PCT/US2013/028608 external-priority patent/WO2013130963A1/en
Publication of JP2015509961A publication Critical patent/JP2015509961A/ja
Publication of JP2015509961A5 publication Critical patent/JP2015509961A5/ja
Application granted granted Critical
Publication of JP6230160B2 publication Critical patent/JP6230160B2/ja
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JP2014560086A 2012-03-02 2013-03-01 Ｉｉｉ型ゴーシェ病を治療するための組成物および方法 Active JP6230160B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261606089P 2012-03-02 2012-03-02
US61/606,089 2012-03-02
PCT/US2013/028608 WO2013130963A1 (en) 2012-03-02 2013-03-01 Compositions and methods for treating type iii gaucher disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2017150619A Division JP6475293B2 (ja) 2012-03-02 2017-08-03 Ｉｉｉ型ゴーシェ病を治療するための組成物および方法

Publications (3)

Publication Number Publication Date
JP2015509961A JP2015509961A (ja) 2015-04-02
JP2015509961A5 true JP2015509961A5 (enExample) 2016-04-21
JP6230160B2 JP6230160B2 (ja) 2017-11-15

Family

ID=49083333

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2014560086A Active JP6230160B2 (ja) 2012-03-02 2013-03-01 Ｉｉｉ型ゴーシェ病を治療するための組成物および方法
JP2017150619A Active JP6475293B2 (ja) 2012-03-02 2017-08-03 Ｉｉｉ型ゴーシェ病を治療するための組成物および方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2017150619A Active JP6475293B2 (ja) 2012-03-02 2017-08-03 Ｉｉｉ型ゴーシェ病を治療するための組成物および方法

Country Status (10)

Country Link
US (1) US9623090B2 (enExample)
EP (1) EP2863941B1 (enExample)
JP (2) JP6230160B2 (enExample)
KR (2) KR20140138850A (enExample)
CN (1) CN104519905A (enExample)
AU (1) AU2013225816B2 (enExample)
CA (1) CA2865614A1 (enExample)
HK (2) HK1207005A1 (enExample)
MX (1) MX361231B (enExample)
WO (1) WO2013130963A1 (enExample)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101658357B1 (ko) 2015-02-17 2016-09-21 한림대학교 산학협력단 레스베라트롤을 함유하는 ⅲ형 고셔병 치료용 약학 조성물
EP3264092A1 (en) * 2016-07-01 2018-01-03 Centogene AG Use of lyso-gb1 as druggable target
PL3672622T3 (pl) 2017-08-24 2024-04-02 Genzyme Corporation Rekombinowana ludzka kwaśna sfingomielinaza do zastosowania w leczeniu nieprawidłowych stanów kości u pacjentów z niedoborem kwaśnej sfingomielinazy
EP3684463B1 (en) 2017-09-19 2025-05-14 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement
EA202091032A1 (ru) 2017-10-26 2020-07-17 Шайр Хьюман Дженетик Терапиз, Инк. Составы, содержащие глюкоцереброзидазу и изофагомин
US11717686B2 (en) 2017-12-04 2023-08-08 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement to facilitate learning and performance
US11318277B2 (en) 2017-12-31 2022-05-03 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement to enhance emotional response
US12280219B2 (en) 2017-12-31 2025-04-22 NeuroLight, Inc. Method and apparatus for neuroenhancement to enhance emotional response
US11364361B2 (en) 2018-04-20 2022-06-21 Neuroenhancement Lab, LLC System and method for inducing sleep by transplanting mental states
US11452839B2 (en) 2018-09-14 2022-09-27 Neuroenhancement Lab, LLC System and method of improving sleep
WO2020157248A1 (en) * 2019-02-01 2020-08-06 Oxyrane Uk Ltd Glucocerebrosidase polypeptides
CN113906012A (zh) * 2019-04-25 2022-01-07 武田药品工业有限公司 异法戈明盐、使用方法和调配物
KR102356047B1 (ko) * 2019-07-26 2022-02-07 재단법인 아산사회복지재단 효소대체요법 치료 비반응성 고셔병 치료용 약학조성물
WO2021183596A1 (en) * 2020-03-10 2021-09-16 University Of Cincinnati Materials and methods for the treatment of gaucher disease

Family Cites Families (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5272066A (en) 1986-03-07 1993-12-21 Massachusetts Institute Of Technology Synthetic method for enhancing glycoprotein stability
US4925796A (en) 1986-03-07 1990-05-15 Massachusetts Institute Of Technology Method for enhancing glycoprotein stability
WO1989005850A1 (en) 1987-12-23 1989-06-29 The United States Of America, As Represented By Th Cloned dna for synthesizing unique glucocerebrosidase
JP2893481B2 (ja) 1988-12-23 1999-05-24 ジェンザイム コーポレイション 酵素的に活性な組換えグルコセレブロシダーゼ
US5549892A (en) 1988-12-23 1996-08-27 Genzyme Corporation Enhanced in vivo uptake of glucocerebrosidase
US5236838A (en) 1988-12-23 1993-08-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
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
EP0568647B1 (en) 1991-01-21 1996-09-11 Genzyme Corporation Production of enzymatically active glucocerebrosidase from recombinant cells
US5641670A (en) 1991-11-05 1997-06-24 Transkaryotic Therapies, Inc. Protein production and protein delivery
US6270989B1 (en) 1991-11-05 2001-08-07 Transkaryotic Therapies, Inc. Protein production and delivery
US5911983A (en) 1992-06-26 1999-06-15 University Of Pittsburgh Gene therapy for Gaucher disease using retroviral vectors
CA2150956A1 (en) 1992-12-10 1994-06-23 Robert G. L. Shorr Glycolipid enzyme-polymer conjugates
WO1994014837A1 (en) 1992-12-21 1994-07-07 Enzon, Inc. Purification of proteinaceous material
EP0675204B1 (en) 1994-03-30 2001-05-30 Takara Shuzo Co. Ltd. Transglycosylating process for producing carbohydrates or glycoconjugates
JP3002113B2 (ja) 1994-03-30 2000-01-24 寳酒造株式会社 糖質又は複合糖質の製造方法
US5670132A (en) 1994-09-20 1997-09-23 Immunomedics, Inc. Modified radioantibody fragments for reduced renal uptake
AU693329B2 (en) 1995-04-13 1998-06-25 Corning Incorporated Dispersion managed optical waveguide
EP0865499B1 (en) 1995-09-14 2009-03-18 Virginia Tech Intellectual Properties, Inc. Production of lysosomal enzymes in plant-based expression systems
WO1997032591A1 (en) 1996-03-05 1997-09-12 Orquest, Inc. Method of promoting bone growth with hyaluronic acid and growth factors
US6177447B1 (en) 1996-07-15 2001-01-23 Universiteit Van Amsterdam Deoxynojirimycin derivatives and their uses as glucosylceramidase inhibitors
NZ506214A (en) 1996-09-13 2002-11-26 Transkaryotic Therapies Inc Therapy for alpha-galactosidase A deficiency
IL155588A0 (en) 2003-04-27 2003-11-23 Metabogal Ltd Methods for expression of enzymatically active recombinant lysosomal enzymes in transgenic plant root cells and vectors used thereby
EP0852951A1 (de) 1996-11-19 1998-07-15 Roche Diagnostics GmbH Stabile lyophilisierte pharmazeutische Zubereitungen von mono- oder polyklonalen Antikörpern
JPH10273500A (ja) 1997-03-03 1998-10-13 Noguchi Inst 複合糖ペプチド及びその製造法
JPH10306099A (ja) 1997-03-04 1998-11-17 Noguchi Inst 新規複合糖ペプチドおよびその製法
TW542721B (en) 1997-08-06 2003-07-21 Melaleuca Inc Dietary supplements containing natural ingredients
DE69825288D1 (de) 1997-08-07 2004-09-02 Univ Utah Prodrugs und konjugate von selenolenthaltenden verbindungen und ihre verwendung
US5939279A (en) 1997-09-18 1999-08-17 The Board Of Trustees Of The University Of Arkansas Inhibition of bacterial binding by high-mannose oligosaccharides
EP1041996A4 (en) 1997-12-22 2003-05-14 Human Genome Sciences Inc KERATINOCYTE GROWTH FACTOR-2 FORMULATIONS
GB9802249D0 (en) 1998-02-03 1998-04-01 Ciba Geigy Ag Organic compounds
GB9807464D0 (en) 1998-04-07 1998-06-10 Pharming Bv Purification of human acid µ-glucosidase
EP0976838A1 (en) 1998-05-06 2000-02-02 Rhone-Poulenc Nutrition Animale Enzymes mixture
JPH11318441A (ja) 1998-05-14 1999-11-24 Nagase & Co Ltd 超耐熱耐酸性アミロプルラナーゼ
KR19990086271A (ko) 1998-05-27 1999-12-15 손경식 면역세포의 신규한 엔도뉴클레아제 및 이를 사용한 면역보조제
KR20010052499A (ko) 1998-06-05 2001-06-25 아방티 파르마 소시에테 아노님 베타-시크리타제형 활성을 지닌 폴리펩타이드
PT1137789E (pt) 1998-12-09 2010-10-21 Phyton Holdings Llc Um método para produzir glicoproteínas possuindo glicosilação de tipo humano
GB9909066D0 (en) 1999-04-20 1999-06-16 Oxford Glycosciences Uk Ltd Therapies
EP1189599A2 (en) 1999-06-11 2002-03-27 Eli Lilly And Company Pharmaceutical materials and methods for their preparation and use
JP2003505430A (ja) 1999-07-26 2003-02-12 ジー・ディー・サール・アンド・カンパニー デオキシノジリマイシンの長鎖ｎ−アルキル誘導体およびグルコセレブロシダーゼ酵素の糖脂質異常蓄積疾患の治療用の薬剤の製造のための使用
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
US6770468B1 (en) 1999-09-14 2004-08-03 Genzyme Glycobiology Research Institute, Inc. Phosphodiester-α-GlcNAcase of the lysosomal targeting pathway
IL150314A0 (en) 1999-12-30 2002-12-01 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
US20040204379A1 (en) 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20020095135A1 (en) 2000-06-19 2002-07-18 David Meeker Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US7138262B1 (en) 2000-08-18 2006-11-21 Shire Human Genetic Therapies, Inc. High mannose proteins and methods of making high mannose proteins
CN1156702C (zh) 2001-07-11 2004-07-07 上海晶泰生物技术有限公司 采用标记链霉亲和素-生物素技术的蛋白质芯片
US20040202666A1 (en) 2003-01-24 2004-10-14 Immunomedics, Inc. Anti-cancer anthracycline drug-antibody conjugates
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
US7951557B2 (en) 2003-04-27 2011-05-31 Protalix Ltd. Human lysosomal proteins from plant cell culture
US20060008415A1 (en) 2004-06-25 2006-01-12 Protein Design Labs, Inc. Stable liquid and lyophilized formulation of proteins
NZ555308A (en) 2004-12-22 2010-10-29 Ambrx Inc Compositions containing, methods involving, and uses of non-natural amino acids and polypeptides
ES2391657T3 (es) 2006-02-07 2012-11-28 Shire Human Genetic Therapies, Inc. Composiciones estabilizadas de proteínas que tienen un resto tiol libre
ES2366059T3 (es) 2006-03-17 2011-10-14 Biomarin Pharmaceutical Inc. Ensayos para la detección de anticuerpos anti-enzimas lisosómicas.
WO2007140212A2 (en) 2006-05-24 2007-12-06 Amicus Therapeutics, Inc. Tartrate salt of isofagomine and methods of use
JP5364362B2 (ja) 2008-12-24 2013-12-11 株式会社城南製作所 ワイヤドラム式のウインドウレギュレータ
AU2010281403B2 (en) 2009-07-28 2016-03-10 Takeda Pharmaceutical Company Limited Compositions and methods for treating Gaucher disease
US20130017169A1 (en) * 2010-03-02 2013-01-17 Protalix Ltd. Multimeric forms of therapeutic proteins and uses thereof
WO2012012461A2 (en) 2010-07-19 2012-01-26 Shire Human Genetic Therapies, Inc. Mannose receptor c type 1 (mrc1) codon optimized cell line and uses thereof

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