JP2013534526A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2013534526A5 JP2013534526A5 JP2013516844A JP2013516844A JP2013534526A5 JP 2013534526 A5 JP2013534526 A5 JP 2013534526A5 JP 2013516844 A JP2013516844 A JP 2013516844A JP 2013516844 A JP2013516844 A JP 2013516844A JP 2013534526 A5 JP2013534526 A5 JP 2013534526A5
- Authority
- JP
- Japan
- Prior art keywords
- disease
- type
- item
- tissue
- enzyme
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
Applications Claiming Priority (15)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US35885710P | 2010-06-25 | 2010-06-25 | |
| US61/358,857 | 2010-06-25 | ||
| US36078610P | 2010-07-01 | 2010-07-01 | |
| US61/360,786 | 2010-07-01 | ||
| US38786210P | 2010-09-29 | 2010-09-29 | |
| US61/387,862 | 2010-09-29 | ||
| US201161435710P | 2011-01-24 | 2011-01-24 | |
| US61/435,710 | 2011-01-24 | ||
| US201161442115P | 2011-02-11 | 2011-02-11 | |
| US61/442,115 | 2011-02-11 | ||
| US201161476210P | 2011-04-15 | 2011-04-15 | |
| US61/476,210 | 2011-04-15 | ||
| US201161495268P | 2011-06-09 | 2011-06-09 | |
| PCT/US2011/041924 WO2011163648A1 (en) | 2010-06-25 | 2011-06-25 | Cns delivery of therapeutic agents |
| US61/495,268 | 2012-06-09 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015236539A Division JP6250616B2 (ja) | 2010-06-25 | 2015-12-03 | 治療薬のcns送達 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013534526A JP2013534526A (ja) | 2013-09-05 |
| JP2013534526A5 true JP2013534526A5 (cg-RX-API-DMAC10.html) | 2014-08-14 |
| JP6045491B2 JP6045491B2 (ja) | 2016-12-14 |
Family
ID=46888845
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013516844A Active JP6045491B2 (ja) | 2010-06-25 | 2011-06-25 | 治療薬のcns送達 |
Country Status (25)
Families Citing this family (50)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2003057179A2 (en) | 2002-01-11 | 2003-07-17 | Biomarin Pharmaceutical, Inc. | Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes |
| CA2803166C (en) | 2010-06-25 | 2023-03-07 | Shire Human Genetic Therapies, Inc. | Cns delivery of therapeutic agents |
| JP6045493B2 (ja) | 2010-06-25 | 2016-12-14 | シャイアー 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 | アリールスルファターゼaのcns送達の方法および組成物 |
| BR112012033214B1 (pt) | 2010-06-25 | 2020-11-03 | Shire Human Genetic Therapies, Inc. | uso de uma formulação estável compreendendo uma proteína de iduronato-2-sulfatase (i2s), formulação compreendendo a mesma e recipiente |
| KR20130043165A (ko) | 2010-06-25 | 2013-04-29 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| JP6073783B2 (ja) | 2010-06-25 | 2017-02-01 | シャイアー 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 | ヘパランn−スルファターゼのcns送達のための方法および組成物 |
| WO2012101671A1 (en) * | 2011-01-25 | 2012-08-02 | Jcr Pharmaceuticals Co., Ltd. | Method for production of human recombinant iduronate 2-sulfatase |
| AU2012278944B2 (en) | 2011-07-05 | 2015-09-17 | Bioasis Technologies Inc. | p97-antibody conjugates and methods of use |
| WO2013055888A2 (en) * | 2011-10-12 | 2013-04-18 | Synageva Biopharma Corp. | Recombinant human naglu protein and uses thereof |
| US9682129B2 (en) | 2011-12-23 | 2017-06-20 | Shire Human Genetic Therapies, Inc. | Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase |
| US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| WO2014022515A1 (en) * | 2012-07-31 | 2014-02-06 | Bioasis Technologies, Inc. | Dephosphorylated lysosomal storage disease proteins and methods of use thereof |
| WO2014110246A1 (en) | 2013-01-09 | 2014-07-17 | Shire Human Genetic Therapies, Inc. | Methods for purification of arylsulfatase a |
| JP2016514096A (ja) | 2013-02-20 | 2016-05-19 | バレリオン セラピューティクス, エルエルシー | ポンぺ病の処置のための方法および組成物 |
| NZ711373A (en) | 2013-03-13 | 2020-07-31 | Bioasis Technologies Inc | Fragments of p97 and uses thereof |
| JP2016523835A (ja) * | 2013-05-15 | 2016-08-12 | リージェンツ オブ ザ ユニバーシティ オブ ミネソタ | 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入 |
| JP6603227B2 (ja) | 2014-02-03 | 2019-11-06 | バイオアシス テクノロジーズ インコーポレイテッド | P97融合タンパク質 |
| AU2015219339B2 (en) | 2014-02-19 | 2020-03-05 | Bioasis Technologies Inc. | P97-IDS fusion proteins |
| CA2943890A1 (en) | 2014-05-01 | 2015-11-05 | Bioasis Technologies, Inc. | P97-polynucleotide conjugates |
| WO2016025519A1 (en) * | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
| AU2015323966A1 (en) | 2014-09-29 | 2017-03-16 | Alexion Pharmaceuticals, Inc. | Methods of treating mucopolysaccharidosis IIIB (MPSIIIB) |
| WO2016100591A1 (en) * | 2014-12-19 | 2016-06-23 | Biogen Ma Inc. | Apparatus for compound dispersion |
| CA2985235A1 (en) | 2015-05-07 | 2016-11-10 | Shire Human Genetic Therapies, Inc. | Glucocerebrosidase gene therapy for parkinson's disease |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| KR20170004814A (ko) * | 2015-07-02 | 2017-01-11 | 주식회사 녹십자 | 헌터증후군 치료제 |
| WO2017062666A2 (en) * | 2015-10-06 | 2017-04-13 | Patricia Dickson | Enzyme replacement therapy for mucopolysaccharidosis iiid |
| US10123969B2 (en) | 2015-10-15 | 2018-11-13 | Wisconsin Alumni Research Foundation | Osmotic enhancement of drug/therapeutic delivery to the brain following infusion or injection into the cerebrospinal fluid |
| SG10201912935WA (en) * | 2015-12-15 | 2020-02-27 | Genzyme Corp | Adeno-associated viral vectors for treating mucolipidosis type ii |
| HK1258054A1 (zh) * | 2015-12-30 | 2019-11-01 | 株式会社绿十字 | 用於治疗亨特综合征的方法和组合物 |
| BR112018016874A2 (pt) * | 2016-02-17 | 2019-02-05 | Shire Human Genetic Therapies, Inc. | métodos e composições para distribuição de arilsulfatase a no sistema nervoso central (snc) |
| US11253612B2 (en) | 2016-04-15 | 2022-02-22 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| CN110621774A (zh) * | 2016-10-14 | 2019-12-27 | 儿童医疗中心有限公司 | 用于治疗中枢神经系统疾病和病症的组合物和方法 |
| CA3050690A1 (en) | 2017-01-17 | 2018-07-26 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for diagnosing and treating peroxisomal diseases |
| GB201709459D0 (en) | 2017-06-14 | 2017-07-26 | Intrabio Ltd | Treatment for migraine |
| WO2019049967A1 (ja) * | 2017-09-07 | 2019-03-14 | Jcrファーマ株式会社 | 水性医薬組成物 |
| EP3684938A1 (en) | 2017-09-22 | 2020-07-29 | The Trustees of the University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| BR112020005271A2 (pt) | 2017-10-02 | 2020-09-15 | Denali Therapeutics Inc. | proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica |
| EP3697406A1 (en) | 2017-10-18 | 2020-08-26 | IntraBio Ltd | Therapeutic agents for improved mobility and cognitive function and for treating neurodegenerative diseases and lysosomal storage disorders |
| US11793782B2 (en) | 2017-10-18 | 2023-10-24 | Intrabio Limited | Therapeutic agents for neurodegenerative diseases |
| CA3086181A1 (en) | 2017-12-19 | 2019-06-27 | Shire Human Genetic Therapies, Inc. | Purified arylsulfatase a and compositons thereof |
| RS65663B1 (sr) | 2018-02-15 | 2024-07-31 | Intrabio Ltd | Acetil leucin za upotrebu u lečenju sindroma nemirnih nogu |
| CN112566652A (zh) | 2018-06-25 | 2021-03-26 | Jcr制药股份有限公司 | 含有蛋白的水性液体制剂 |
| CN119097618A (zh) | 2019-03-02 | 2024-12-10 | 内在生物技术有限公司 | 用于治疗疾病的亮氨酸、乙酰基亮氨酸和相关类似物 |
| MD3989962T2 (ro) | 2019-06-28 | 2025-12-31 | Intrabio Ltd Gb | Terapie combinată cu acetil-leucină și miglustat pentru tratamentul unei boli de depozitare lizozomală |
| EP4438120A3 (en) * | 2019-08-29 | 2024-12-11 | BioMarin Pharmaceutical Inc. | Methods for treating cln2 disease in pediatric subjects |
| WO2021144720A1 (en) | 2020-01-13 | 2021-07-22 | Intrabio Ltd | Treatment of late-onset neurodegenerative diseases in heterozygous npc1 gene mutation carriers |
| KR20230015433A (ko) | 2020-05-22 | 2023-01-31 | 인트라바이오 리미티드 | 운동실조를 치료하기 위한 아세틸 류신과 4-아미노피리딘 또는 아세타졸아미드의 조합물 |
| CN120641109A (zh) * | 2022-11-09 | 2025-09-12 | Gmp生物技术有限公司 | 用于cns疾病的组合物和方法 |
| WO2024176112A1 (en) | 2023-02-21 | 2024-08-29 | Takeda Pharmaceutical Company Limited | Large scale production of recombinant arylsulfatase a and compositions thereof |
| TW202604470A (zh) | 2024-06-20 | 2026-02-01 | 美商音特拉拜歐公司 | 乙醯白胺酸用於治療神經發育障礙 |
Family Cites Families (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4743265A (en) * | 1986-04-23 | 1988-05-10 | Dij Catheter Corp | Articulated catheter placement device |
| US5863782A (en) | 1995-04-19 | 1999-01-26 | Women's And Children's Hospital | Synthetic mammalian sulphamidase and genetic sequences encoding same |
| US6118045A (en) | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
| ES2185117T3 (es) | 1997-08-22 | 2003-04-16 | Seikagaku Kogyo Co Ltd | Agente terapeutico para disco intervertebral herniado. |
| DK2020438T3 (en) | 1998-12-07 | 2018-07-23 | Genzyme Corp | Treatment of Pompe disease |
| US6217552B1 (en) | 1999-03-01 | 2001-04-17 | Coaxia, Inc. | Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma |
| US20020052311A1 (en) | 1999-09-03 | 2002-05-02 | Beka Solomon | Methods and compostions for the treatment and/or diagnosis of neurological diseases and disorders |
| US6534300B1 (en) | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
| US20020099025A1 (en) * | 1999-12-30 | 2002-07-25 | Heywood James A. | Treatment of neurological disorders |
| WO2003102583A1 (en) | 2002-05-29 | 2003-12-11 | Symbiontics, Inc. | Targeted therapeutic proteins |
| EP1436316B1 (en) | 2001-04-30 | 2008-01-23 | ZyStor Therapeutics , Inc. | Subcellular targeting of therapeutic proteins |
| US20040005309A1 (en) | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| WO2003032913A2 (en) | 2001-10-16 | 2003-04-24 | Symbiontics Inc. | Methods and compositions for targeting proteins across the blood brain barrier |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| DK1503788T3 (da) * | 2002-04-25 | 2011-10-17 | Shire Human Genetic Therapies | Behandling af alpha-galactosidase A-deficiens |
| PT1587923E (pt) | 2003-01-22 | 2011-12-07 | Univ Duke | Construções melhoradas para a expressão de polipéptidos lisossomais |
| US20050026823A1 (en) * | 2003-06-20 | 2005-02-03 | Biomarin Pharmaceutical Inc. | Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues |
| WO2005002515A2 (en) | 2003-06-20 | 2005-01-13 | 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 |
| NZ576986A (en) | 2004-01-30 | 2010-12-24 | Shire Pharmaceuticals Ireland Ltd | Production and purification of recombinant arylsulfatase A |
| WO2005074888A2 (en) | 2004-02-03 | 2005-08-18 | Biodelivery Sciences International, Inc. | Replacement enzyme cochleates |
| US20080014188A1 (en) | 2004-02-06 | 2008-01-17 | Zankel Todd C | Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof |
| DE602005020745D1 (de) | 2004-02-10 | 2010-06-02 | Zystor Therapeutics Inc | Saure alpha-glukosidase und fragmente davon |
| US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
| EP1889628B1 (en) | 2005-05-11 | 2013-12-11 | JCR PHARMACEUTICALS Co., LTD. | Lipid liposome composition |
| ES2485369T3 (es) | 2005-06-08 | 2014-08-13 | Amicus Therapeutics, Inc. | Tratamiento de trastornos del SNC asociados con mutaciones en genes que codifican enzimas lisosómicas |
| AR059089A1 (es) * | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| NO347673B1 (no) * | 2006-04-04 | 2024-02-19 | Takeda Pharmaceuticals Co | En sammensetning som omfatter konsentrert polypeptide og bruk derav. |
| GB0611463D0 (en) | 2006-06-09 | 2006-07-19 | Novartis Ag | Organic compounds |
| US8419710B2 (en) * | 2006-12-06 | 2013-04-16 | Medtronic, Inc. | Methods for infusing fluids via an implantable infusion system |
| WO2008109677A2 (en) | 2007-03-06 | 2008-09-12 | Saint Louis University | Modified enzyme and treatment method |
| JP5341756B2 (ja) * | 2007-06-29 | 2013-11-13 | 国立大学法人名古屋大学 | 神経障害に基づく機能不全の改善剤およびRhoキナーゼ活性化抑制剤 |
| WO2009017005A1 (ja) | 2007-07-27 | 2009-02-05 | Sharp Kabushiki Kaisha | 移動局装置、基地局装置、通信システム及びプログラム |
| CA2695374A1 (en) | 2007-08-15 | 2009-02-19 | Amunix, Inc. | Compositions and methods for modifying properties of biologically active polypeptides |
| US8420081B2 (en) | 2007-11-30 | 2013-04-16 | Abbvie, Inc. | Antibody formulations and methods of making same |
| US7722865B2 (en) | 2008-01-18 | 2010-05-25 | Biomarin Pharmaceutical Inc. | Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof |
| WO2009137721A2 (en) | 2008-05-07 | 2009-11-12 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides and uses thereof |
| US8436489B2 (en) | 2009-06-29 | 2013-05-07 | Lightsail Energy, Inc. | Compressed air energy storage system utilizing two-phase flow to facilitate heat exchange |
| JP6073783B2 (ja) | 2010-06-25 | 2017-02-01 | シャイアー 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 | ヘパランn−スルファターゼのcns送達のための方法および組成物 |
| CA2803166C (en) | 2010-06-25 | 2023-03-07 | Shire Human Genetic Therapies, Inc. | Cns delivery of therapeutic agents |
| JP6045493B2 (ja) | 2010-06-25 | 2016-12-14 | シャイアー 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 | アリールスルファターゼaのcns送達の方法および組成物 |
| CN103269709A (zh) | 2010-06-25 | 2013-08-28 | 夏尔人类遗传性治疗公司 | Sanfilippo综合症类型B的治疗 |
| KR20130043165A (ko) | 2010-06-25 | 2013-04-29 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| BR112012033214B1 (pt) | 2010-06-25 | 2020-11-03 | Shire Human Genetic Therapies, Inc. | uso de uma formulação estável compreendendo uma proteína de iduronato-2-sulfatase (i2s), formulação compreendendo a mesma e recipiente |
| EP2588132A4 (en) | 2010-06-25 | 2014-10-15 | Shire Human Genetic Therapies | METHOD AND COMPOSITIONS FOR DELIVERING BETA GALACTOCEREBROSIDASE INTO THE CNS |
| JP2012062312A (ja) | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | ハンター症候群の治療剤 |
-
2011
- 2011-06-25 CA CA2803166A patent/CA2803166C/en active Active
- 2011-06-25 KR KR1020217042861A patent/KR102663407B1/ko active Active
- 2011-06-25 EP EP11799035.8A patent/EP2588130B1/en active Active
- 2011-06-25 AU AU2011270668A patent/AU2011270668B8/en active Active
- 2011-06-25 US US13/168,961 patent/US9283181B2/en active Active
- 2011-06-25 EP EP20212573.8A patent/EP3875107A1/en active Pending
- 2011-06-25 RU RU2012154831A patent/RU2626514C2/ru active
- 2011-06-25 ME MEP-2016-249A patent/ME02549B/me unknown
- 2011-06-25 MX MX2018003131A patent/MX389903B/es unknown
- 2011-06-25 KR KR1020137001748A patent/KR102007044B1/ko active Active
- 2011-06-25 ES ES11799035T patent/ES2650689T3/es active Active
- 2011-06-25 DK DK11799035.8T patent/DK2588130T3/en active
- 2011-06-25 EP EP16179150.4A patent/EP3103469B1/en not_active Revoked
- 2011-06-25 RS RS20160974A patent/RS55533B1/sr unknown
- 2011-06-25 LT LTEP11799035.8T patent/LT2588130T/lt unknown
- 2011-06-25 JP JP2013516844A patent/JP6045491B2/ja active Active
- 2011-06-25 KR KR1020217023503A patent/KR102345973B1/ko active Active
- 2011-06-25 SI SI201131018A patent/SI2588130T1/sl unknown
- 2011-06-25 CN CN201180040978.5A patent/CN103096918B/zh active Active
- 2011-06-25 SM SM20160385T patent/SMT201600385T1/it unknown
- 2011-06-25 PL PL16179150T patent/PL3103469T3/pl unknown
- 2011-06-25 UA UAA201214666A patent/UA115648C2/uk unknown
- 2011-06-25 PE PE2022001782A patent/PE20230169A1/es unknown
- 2011-06-25 WO PCT/US2011/041924 patent/WO2011163648A1/en not_active Ceased
- 2011-06-25 KR KR1020217003036A patent/KR102283671B1/ko active Active
- 2011-06-25 KR KR1020247014519A patent/KR20240068752A/ko not_active Withdrawn
- 2011-06-25 IL IL291554A patent/IL291554B2/en unknown
- 2011-06-25 NZ NZ605865A patent/NZ605865A/en unknown
- 2011-06-25 HR HRP20161505TT patent/HRP20161505T1/hr unknown
- 2011-06-25 KR KR1020197022037A patent/KR102211960B1/ko active Active
-
2012
- 2012-12-18 IL IL223717A patent/IL223717B/en active IP Right Grant
-
2013
- 2013-01-25 ZA ZA2013/00671A patent/ZA201300671B/en unknown
-
2016
- 2016-11-17 CY CY20161101188T patent/CY1118265T1/el unknown
-
2017
- 2017-04-10 AU AU2017202349A patent/AU2017202349B2/en active Active
-
2019
- 2019-06-11 AU AU2019204056A patent/AU2019204056B2/en active Active
-
2020
- 2020-04-07 IL IL273889A patent/IL273889B/en unknown
-
2021
- 2021-03-08 CY CY20211100195T patent/CY1124154T1/el unknown
- 2021-10-20 AU AU2021254564A patent/AU2021254564B2/en active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| RU2012154831A (ru) | Доставка к цнс лечебных агентов | |
| US12161702B2 (en) | Methods and compositions for CNS delivery of heparan N-sulfatase | |
| Parenti et al. | Lysosomal storage diseases: from pathophysiology to therapy | |
| US20210228692A1 (en) | Intraventricular enzyme delivery for lysosomal storage diseases | |
| US9320711B2 (en) | Methods and compositions for CNS delivery of heparan N-sulfatase | |
| EP2585105B1 (en) | Treatment of sanfilippo syndrome type b | |
| KR102356192B1 (ko) | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 | |
| US20110213328A1 (en) | Methods and Systems for Treatment of Neurological Diseases of the Central Nervous System | |
| US20170216412A1 (en) | Recombinant human naglu protein and uses thereof | |
| Dunder et al. | Enzyme replacement therapy in a mouse model of aspartylglycosaminuria | |
| US20180071401A1 (en) | Intranasal Administration of Guanidinylated Aminoglycosides | |
| Balan | Lysosomal Storage Disorders: New Therapies in the Horizon | |
| HK40059834A (en) | Pharmaceutical formulation comprising a replacement enzyme for a lysosomal enzyme for use in treating lysosomal storage disease intrathecally | |
| HK1230954B (en) | Cns delivery of therapeutic agents | |
| HK1184712B (en) | Cns delivery of therapeutic agents | |
| HK1230954A1 (en) | Cns delivery of therapeutic agents | |
| TR201808258T4 (tr) | Alfa-mannosidazın tıbbi kullanımı. | |
| NZ623690B2 (en) | Recombinant human naglu protein and uses thereof |