JP6063380B2 - サンフィリポ症候群ｂ型の処置 - Google Patents
サンフィリポ症候群ｂ型の処置 Download PDFInfo
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| PCT/US2011/041928 WO2011163652A2 (en) | 2010-06-25 | 2011-06-25 | Treatment of sanfilippo syndrome type b |
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| WO2008022349A2 (en) | 2006-08-18 | 2008-02-21 | Armagen Technologies, Inc. | Agents for blood-brain barrier delivery |
| EP2997976A1 (en) | 2007-07-27 | 2016-03-23 | Armagen Technologies, Inc. | Methods and compositions for increasing alpha-l-iduronidase activity in the cns |
| CA2723412A1 (en) * | 2008-05-07 | 2009-11-12 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides and uses thereof |
| HRP20190690T1 (hr) | 2009-10-09 | 2019-06-28 | Armagen, Inc. | Postupci i pripravci za povećanje aktiviteta iduronat-2-sulfataze u centralnom nervnom sustavu |
| SI2585104T1 (sl) | 2010-06-25 | 2019-11-29 | Shire Human Genetic Therapies | Postopki in sestavki za dostavo arilsulfataze A |
| HUE046856T2 (hu) | 2010-06-25 | 2020-03-30 | Shire Human Genetic Therapies | Eljárások és kompozíciók iduronát-2-szulfatáz központi idegrendszerbe történõ leadásához |
| SMT201600385T1 (it) | 2010-06-25 | 2017-03-08 | Shire Human Genetic Therapies | Trasporto di agenti terapeutici all’snc |
| CN103260637B (zh) | 2010-06-25 | 2016-04-06 | 夏尔人类遗传性治疗公司 | 乙酰肝素n-硫酸酯酶cns递送的方法和组合物 |
| EP2975134A3 (en) * | 2010-07-22 | 2016-02-24 | Shire Human Genetic Therapies, Inc. | Crystal structure of human alpha-n-acetylglucosaminidase |
| WO2012122042A2 (en) * | 2011-03-04 | 2012-09-13 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| SG11201401457SA (en) * | 2011-10-12 | 2014-05-29 | Synageva Biopharma Corp | Recombinant human naglu protein and uses thereof |
| EP4338797A3 (en) | 2011-12-02 | 2024-06-12 | Armagen, Inc. | Methods and compositions for increasing arylsulfatase a activity in the cns |
| WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| KR101380740B1 (ko) * | 2012-06-29 | 2014-04-11 | 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 | 이듀로네이트-2-설파타제의 정제 |
| HUE043679T2 (hu) * | 2012-11-27 | 2019-09-30 | Biomarin Pharm Inc | Célra irányított terápiás lizoszomális enzim fúziós fehérjék és alkalmazásaik |
| FR3011468B1 (fr) * | 2013-10-04 | 2015-12-04 | Inventiva | Utilisation de l'odiparcil dans le traitement d'une mucopolysaccharidose |
| EP3160494B1 (en) * | 2014-06-25 | 2019-11-20 | Shire Human Genetic Therapies, Inc. | Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase |
| MA40460A (fr) | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies | Ciblage lysosomial et utilisation correspondante |
| US10722559B2 (en) | 2014-08-11 | 2020-07-28 | Shire Human Genetic Therapies, Inc. | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
| US10413597B2 (en) | 2014-09-29 | 2019-09-17 | Alexion Pharmaceuticals, Inc. | Methods of treating Mucopolysaccharidosis IIIB (MPSIIIB) |
| WO2016065319A1 (en) * | 2014-10-24 | 2016-04-28 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting of enzymes, and uses thereof |
| MA41022A (fr) | 2014-11-24 | 2017-10-03 | Shire Human Genetic Therapies | Ciblage lysosomial et utilisations correspondantes |
| US10538589B2 (en) * | 2015-01-14 | 2020-01-21 | Armagen Inc. | Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU |
| US11345904B2 (en) | 2016-02-24 | 2022-05-31 | Biomarin Pharmaceutical Inc. | Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof |
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| CN116234901A (zh) * | 2020-08-28 | 2023-06-06 | Jcr制药股份有限公司 | α-N-乙酰氨基葡萄糖苷酶的突变体 |
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| US6118045A (en) | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
| ATE410506T1 (de) | 1998-12-07 | 2008-10-15 | Genzyme Corp | Behandlung der pompeschen krankheit |
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| WO2002087510A2 (en) | 2001-04-30 | 2002-11-07 | Symbiontics, Inc. | Subcellular targeting of therapeutic proteins |
| US20040005309A1 (en) | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
| US7560424B2 (en) * | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| IL161352A0 (en) | 2001-10-16 | 2004-09-27 | Symbiontics Inc | Methods and compositions for targeting underglycosylated proteins across the blood brain barrier |
| CA2487815A1 (en) | 2002-05-29 | 2003-12-11 | Symbiontics, Inc. | Targeted therapeutic proteins |
| PT1587923E (pt) | 2003-01-22 | 2011-12-07 | Univ Duke | Construções melhoradas para a expressão de polipéptidos lisossomais |
| CA2525236C (en) * | 2003-06-20 | 2015-03-24 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US7442372B2 (en) * | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US20050244400A1 (en) | 2004-02-10 | 2005-11-03 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| GB0611463D0 (en) * | 2006-06-09 | 2006-07-19 | Novartis Ag | Organic compounds |
| WO2008109677A2 (en) | 2007-03-06 | 2008-09-12 | Saint Louis University | Modified enzyme and treatment method |
| CA2723412A1 (en) | 2008-05-07 | 2009-11-12 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides and uses thereof |
| CN103260637B (zh) * | 2010-06-25 | 2016-04-06 | 夏尔人类遗传性治疗公司 | 乙酰肝素n-硫酸酯酶cns递送的方法和组合物 |
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| AU2011270672A1 (en) | 2013-02-14 |
| US20110318327A1 (en) | 2011-12-29 |
| NZ605871A (en) | 2015-02-27 |
| EP2585105A2 (en) | 2013-05-01 |
| WO2011163652A2 (en) | 2011-12-29 |
| CA2805449A1 (en) | 2011-12-29 |
| KR20130043166A (ko) | 2013-04-29 |
| EP2585105B1 (en) | 2017-08-09 |
| JP2013542913A (ja) | 2013-11-28 |
| CN103269709A (zh) | 2013-08-28 |
| AU2017202344A1 (en) | 2017-04-27 |
| EP2585105A4 (en) | 2014-07-09 |
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