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
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pharmaceutical composition
subject
replacement therapy
gaucher disease
enzyme replacement
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JP2014560086A
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JP6230160B2 (ja
JP2015509961A (ja
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Priority claimed from PCT/US2013/028608 external-priority patent/WO2013130963A1/en
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Publication of JP2015509961A5 publication Critical patent/JP2015509961A5/ja
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Claims (14)

  1. グルコセレブロシダーゼ酵素補充療法薬を含む、III型ゴーシェ病に罹患する対象を治療するための医薬組成物であって、前記グルコセレブロシダーゼ酵素補充療法薬が、前記対象2時間未満にわた静脈内注入される医薬組成物
  2. グルコセレブロシダーゼ酵素補充療法は、90分以内にわたって投与される、請求項1記載の医薬組成物
  3. グルコセレブロシダーゼ酵素補充療法は、60分以内にわたって投与される、請求項1記載の医薬組成物
  4. 前記対象への2時間未満にわたる静脈内注入によって投与される第2のグルコセレブロシダーゼ酵素補充療法薬と組み合わせて投与される、請求項1記載の医薬組成物
  5. 前記グルコセレブロシダーゼ酵素補充療法は、ベラグルセラーゼ、イミグルセラーゼ、およびuplyso(商標)から成る群より選択される、請求項1記載の医薬組成物
  6. 前記グルコセレブロシダーゼ酵素補充療法は、15〜60U/kgの用量で投与される、請求項1記載の医薬組成物
  7. グルコセレブロシダーゼ酵素補充療法を現在受けているかまたは以前受けたことがあるIII型ゴーシェ病に罹患する対象を治療するための医薬組成物であって、ベラグルセラーゼを含み、前記対象が、ゴーシェ病のために現在受けているまたは以前に受けたことがある療法に対する抗体の産生の検査で陽性反応を示したことに基づいて選択される医薬組成物
  8. 前記対象が、ゴーシェ病のために現在受けているまたは以前に受けたことがある療法に対するIgE抗体の産生の検査で陽性反応を示す、請求項記載の医薬組成物
  9. 前記対象が、ゴーシェ病のために現在受けているまたは以前に受けたことがある療法に対するIgM抗体の産生の検査で陽性反応を示す、請求項記載の医薬組成物
  10. 前記対象が、ゴーシェ病のために現在受けているまたは以前に受けたことがある療法に対するIgG抗体の産生の検査で陽性反応を示す、請求項記載の医薬組成物
  11. 前記対象がゴーシェ病のために現在受けているまたは以前に受けたことがある療法が、イミグルセラーゼである、請求項記載の医薬組成物
  12. 前記対象がゴーシェ病のために現在受けているまたは以前に受けたことがある療法が、uplyso(商標)である、請求項記載の医薬組成物
  13. ベラグルセラーゼが、15〜60U/kgの用量で投与される、請求項記載の医薬組成物
  14. 前記ベラグルセラーゼが、90分以内にわたって静脈内注入により前記対象に投与される、請求項記載の医薬組成物
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)

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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 (ja) 2016-04-21
JP6230160B2 JP6230160B2 (ja) 2017-11-15

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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)

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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 (ja)
EP (1) EP2863941B1 (ja)
JP (2) JP6230160B2 (ja)
KR (2) KR20140138850A (ja)
CN (1) CN104519905A (ja)
AU (1) AU2013225816B2 (ja)
CA (1) CA2865614A1 (ja)
HK (2) HK1207005A1 (ja)
MX (1) MX361231B (ja)
WO (1) WO2013130963A1 (ja)

Families Citing this family (12)

* 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
JP7216075B2 (ja) 2017-08-24 2023-01-31 サノフイ 酸性スフィンゴミエリナーゼ欠損症患者における骨状態異常の処置
US11723579B2 (en) 2017-09-19 2023-08-15 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement
JP2021501135A (ja) * 2017-10-26 2021-01-14 シャイア ヒューマン ジェネティック セラピーズ インコーポレイテッド グルコセレブロシダーゼ及びイソファゴミンを含む製剤
US11717686B2 (en) 2017-12-04 2023-08-08 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement to facilitate learning and performance
EP3731749A4 (en) 2017-12-31 2022-07-27 Neuroenhancement Lab, LLC NEURO-ACTIVATION SYSTEM AND METHOD FOR ENHANCING EMOTIONAL RESPONSE
US11364361B2 (en) 2018-04-20 2022-06-21 Neuroenhancement Lab, LLC System and method for inducing sleep by transplanting mental states
CN113382683A (zh) 2018-09-14 2021-09-10 纽罗因恒思蒙特实验有限责任公司 改善睡眠的系统和方法
EP3917558A1 (en) * 2019-02-01 2021-12-08 Oxyrane UK Limited Glucocerebrosidase polypeptides
KR102356047B1 (ko) * 2019-07-26 2022-02-07 재단법인 아산사회복지재단 효소대체요법 치료 비반응성 고셔병 치료용 약학조성물
EP4117628A4 (en) * 2020-03-10 2024-04-10 University of Cincinnati MATERIALS AND METHODS FOR THE TREATMENT OF GAUCHER’S DISEASE

Family Cites Families (59)

* 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
AU2946289A (en) 1987-12-23 1989-07-19 United States of America, as represented by the Secretary, U.S. Department of Commerce, The Cloned dna for synthesizing unique glucocerebrosidase
ES2093642T3 (es) 1988-12-23 1997-01-01 Genzyme Corp Celulas cho que producen gluco-cerebrosidasa re-combinante enzimaticamente activa.
US5236838A (en) 1988-12-23 1993-08-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5549892A (en) 1988-12-23 1996-08-27 Genzyme Corporation Enhanced in vivo uptake of glucocerebrosidase
JPH03503721A (ja) 1988-12-23 1991-08-22 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
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
CA2099876C (en) 1991-01-21 2002-03-26 Michael L. Hayes Production of enzymatically active glucocerebrosidase from recombinant cells
US6270989B1 (en) 1991-11-05 2001-08-07 Transkaryotic Therapies, Inc. Protein production and delivery
US5641670A (en) 1991-11-05 1997-06-24 Transkaryotic Therapies, Inc. Protein production and protein delivery
US5911983A (en) 1992-06-26 1999-06-15 University Of Pittsburgh Gene therapy for Gaucher disease using retroviral vectors
AU5743994A (en) 1992-12-10 1994-07-04 Enzon, Inc. Glycolipid enzyme-polymer conjugates
WO1994014837A1 (en) 1992-12-21 1994-07-07 Enzon, Inc. Purification of proteinaceous material
DE69521074T2 (de) 1994-03-30 2001-09-13 Takara Shuzo Co Transglykosylierungsverfahren zur Herstellung eines Kohlenhydrats oder eines Glykokonjugates
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
US5929304A (en) 1995-09-14 1999-07-27 Croptech Development Corporation Production of lysosomal enzymes in plant-based expression systems
JP2002504083A (ja) 1996-03-05 2002-02-05 オーケスト インコーポレイテッド ヒアルロン酸および増殖因子による骨の増殖を促進する方法
EP0912179B1 (en) 1996-07-15 2006-04-12 MacroZyme DNM B.V. Deoxynojirimycin derivatives and their uses as glucosylceramidase inhibitors
AU4424497A (en) 1996-09-13 1998-04-02 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
DE69835308T2 (de) 1997-08-07 2007-08-09 University Of Utah Research Foundation, Salt Lake City Prodrugs und Konjugate von Selenoenthaltenden 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
CA2316015A1 (en) 1997-12-22 1999-07-01 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
ES2558160T3 (es) 1998-12-09 2016-02-02 Phyton Holdings, Llc Glicoproteínas que tienen glicosilación de tipo humano
GB9909066D0 (en) 1999-04-20 1999-06-16 Oxford Glycosciences Uk Ltd Therapies
WO2000076480A2 (en) 1999-06-11 2000-12-21 Eli Lilly And Company Pharmaceutical materials and methods for their preparation and use
ATE234626T1 (de) 1999-07-26 2003-04-15 Searle & Co Verwendung von langkettigen n-alkyl derivaten von deoxynojirimycin mit glucocerebrosidase enzyme zur herstellung eines medikaments zur behandlung von mit glykolipiden akkumulation zusammenhängenden krankheiten
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
AU2352201A (en) 1999-12-30 2001-07-16 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
WO2001097829A2 (en) 2000-06-19 2001-12-27 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20040204379A1 (en) 2000-06-19 2004-10-14 Cheng Seng H. 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
ES2619353T3 (es) 2003-01-31 2017-06-26 Mount Sinai School Of Medicine Of New York University Terapia de combinación para tratar trastornos de deficiencia de proteínas
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
WO2006069246A2 (en) 2004-12-22 2006-06-29 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.
DE602007013648D1 (de) 2006-05-24 2011-05-19 Amicus Therapeutics Inc Tartratsalz aus Isofagomin und Verwendungsverfahren dafür
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
RU2733466C2 (ru) * 2009-07-28 2020-10-01 Шайр Хьюман Дженетик Терапиз Композиции и способы для лечения болезни гоше
WO2011107992A2 (en) 2010-03-02 2011-09-09 Protalix Ltd. Multimeric forms of therapeutic proteins and uses thereof
US9453847B2 (en) 2010-07-19 2016-09-27 Shire Human Genetic Therapies, Inc. Mannose receptor C type 1 (MRC1) codon optimized cell line and uses thereof

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