JP2007504166A5 - - Google Patents

Download PDF

Info

Publication number
JP2007504166A5
JP2007504166A5 JP2006524936A JP2006524936A JP2007504166A5 JP 2007504166 A5 JP2007504166 A5 JP 2007504166A5 JP 2006524936 A JP2006524936 A JP 2006524936A JP 2006524936 A JP2006524936 A JP 2006524936A JP 2007504166 A5 JP2007504166 A5 JP 2007504166A5
Authority
JP
Japan
Prior art keywords
pharmaceutical composition
composition according
disease
type
cell
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
Application number
JP2006524936A
Other languages
English (en)
Japanese (ja)
Other versions
JP5819574B2 (ja
JP2007504166A (ja
Filing date
Publication date
Priority claimed from US10/651,493 external-priority patent/US7442372B2/en
Application filed filed Critical
Publication of JP2007504166A publication Critical patent/JP2007504166A/ja
Publication of JP2007504166A5 publication Critical patent/JP2007504166A5/ja
Application granted granted Critical
Publication of JP5819574B2 publication Critical patent/JP5819574B2/ja
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

Links

JP2006524936A 2003-08-29 2004-08-30 脳およびその他の組織への治療用化合物の送達 Expired - Lifetime JP5819574B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US10/651,493 US7442372B2 (en) 2003-08-29 2003-08-29 Delivery of therapeutic compounds to the brain and other tissues
US10/651,493 2003-08-29
PCT/US2004/028135 WO2005021064A2 (en) 2003-08-29 2004-08-30 Delivery of therapeutic compounds to the brain and other tissues

Publications (3)

Publication Number Publication Date
JP2007504166A JP2007504166A (ja) 2007-03-01
JP2007504166A5 true JP2007504166A5 (https=) 2012-10-18
JP5819574B2 JP5819574B2 (ja) 2015-11-24

Family

ID=34217412

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2006524936A Expired - Lifetime JP5819574B2 (ja) 2003-08-29 2004-08-30 脳およびその他の組織への治療用化合物の送達

Country Status (12)

Country Link
US (5) US7442372B2 (https=)
EP (1) EP1673104B2 (https=)
JP (1) JP5819574B2 (https=)
AU (1) AU2004268645B2 (https=)
CA (1) CA2537238C (https=)
CY (1) CY1114815T1 (https=)
DK (1) DK1673104T4 (https=)
ES (1) ES2441612T5 (https=)
PL (1) PL1673104T5 (https=)
PT (1) PT1673104E (https=)
SI (1) SI1673104T2 (https=)
WO (1) WO2005021064A2 (https=)

Families Citing this family (89)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6585971B1 (en) * 1999-11-12 2003-07-01 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof
US7723296B2 (en) * 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
JP2008503590A (ja) * 2004-06-21 2008-02-07 メドトロニック・インコーポレーテッド 組成物を細胞にデリバリーするための医学用システム及び方法
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
DK1988823T3 (en) 2006-02-09 2018-12-03 Genzyme Corp SLOW INTRAVENTRICULAR ADMINISTRATION
US20080015546A1 (en) * 2006-07-13 2008-01-17 Casas Jesus W Methods and formulations for optimal local delivery of cell therapy via minimally invasive procedures
US8849372B2 (en) * 2006-11-22 2014-09-30 The General Hospital Corporation Method for magnetic resonance imaging using stimulus induced rotary saturation with a contrast agent
WO2008070769A1 (en) * 2006-12-06 2008-06-12 Medtronic, Inc. Intrathecal catheter
SI2457920T1 (en) 2007-01-18 2018-02-28 Genzyme Corporation Oligosaccharides containing the amino acid group and their conjugates
WO2008089339A2 (en) * 2007-01-18 2008-07-24 Genzyme Corporation Oligosaccharide conjugates for cellular targeting
PL3252161T3 (pl) * 2007-06-06 2022-03-07 Genzyme Corporation Terapia genowa dla lizosomalnych chorób spichrzeniowych
US8162924B2 (en) 2007-08-17 2012-04-24 The Invention Science Fund I, Llc System, devices, and methods including actively-controllable superoxide water generating systems
US20090048648A1 (en) * 2007-08-17 2009-02-19 Searete Llc, A Limited Liability Corporation Of The State Of Delaware Self-sterilizing device
US8706211B2 (en) 2007-08-17 2014-04-22 The Invention Science Fund I, Llc Systems, devices, and methods including catheters having self-cleaning surfaces
US8702640B2 (en) 2007-08-17 2014-04-22 The Invention Science Fund I, Llc System, devices, and methods including catheters configured to monitor and inhibit biofilm formation
US8460229B2 (en) 2007-08-17 2013-06-11 The Invention Science Fund I, Llc Systems, devices, and methods including catheters having components that are actively controllable between transmissive and reflective states
US8114346B2 (en) * 2007-08-17 2012-02-14 The Invention Science Fund I, Llc Event-triggered ultraviolet light sterilization of surfaces
US8753304B2 (en) 2007-08-17 2014-06-17 The Invention Science Fund I, Llc Systems, devices, and methods including catheters having acoustically actuatable waveguide components for delivering a sterilizing stimulus to a region proximate a surface of the catheter
US8366652B2 (en) 2007-08-17 2013-02-05 The Invention Science Fund I, Llc Systems, devices, and methods including infection-fighting and monitoring shunts
US8029740B2 (en) 2008-07-11 2011-10-04 The Invention Science Fund I, Llc Event-triggered self-sterilization of article surfaces
US8734718B2 (en) 2007-08-17 2014-05-27 The Invention Science Fund I, Llc Systems, devices, and methods including catheters having an actively controllable therapeutic agent delivery component
US8647292B2 (en) 2007-08-17 2014-02-11 The Invention Science Fund I, Llc Systems, devices, and methods including catheters having components that are actively controllable between two or more wettability states
EP2192924B1 (en) * 2007-08-20 2017-10-11 Protalix Ltd. Saccharide-containing protein conjugates and uses thereof
EP2219666A4 (en) * 2007-11-15 2011-05-25 Univ North Carolina PROSTATE ACID PHOSPHATASE FOR THE TREATMENT OF PAIN
HRP20170698T1 (hr) 2008-05-07 2017-09-22 Biomarin Pharmaceutical Inc. Lizosomski usmjereni peptidi i njihove uporabe
US20120041287A1 (en) 2008-12-04 2012-02-16 Searete Llc, A Limited Liability Corporation Of The State Of Delaware Systems, devices, and methods including implantable devices with anti-microbial properties
EP2384168B1 (en) 2008-12-04 2014-10-08 Searete LLC Actively-controllable sterilizing excitation delivery implants
US8585627B2 (en) 2008-12-04 2013-11-19 The Invention Science Fund I, Llc Systems, devices, and methods including catheters configured to monitor biofilm formation having biofilm spectral information configured as a data structure
CA3040973A1 (en) 2008-12-16 2010-07-01 Genzyme Corporation Oligosaccharide-protein conjugates
EP2475376B1 (en) 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
SI3998078T1 (sl) * 2009-08-28 2026-02-27 Genzyme Corporation Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze
US9194011B2 (en) 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
NZ605873A (en) * 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of arylsulfatase a
EP2588132A4 (en) * 2010-06-25 2014-10-15 Shire Human Genetic Therapies METHOD AND COMPOSITIONS FOR DELIVERING BETA GALACTOCEREBROSIDASE INTO THE CNS
RS62620B1 (sr) * 2010-06-25 2021-12-31 Shire Human Genetic Therapies Metode i kompozicije za isporuku cns akrilsulfataze a
SMT201600385T1 (it) * 2010-06-25 2017-03-08 Shire Human Genetic Therapies Trasporto di agenti terapeutici all’snc
NZ605874A (en) * 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of heparan n-sulfatase
CN103179980B (zh) * 2010-06-25 2016-09-28 夏尔人类遗传性治疗公司 艾杜糖醛酸-2-硫酸酯酶的cns递送的方法和组合物
CA2805449A1 (en) * 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
JP2012062312A (ja) * 2010-08-19 2012-03-29 Yoshikatsu Eto ハンター症候群の治療剤
CN103443270B (zh) 2011-01-20 2017-06-06 普罗塔里克斯有限公司 用于在植物和植物细胞中表达α‑半乳糖苷酶的核酸构建体
JP6329483B2 (ja) * 2011-10-12 2018-05-23 シナジェバ・バイオファーマ・コーポレイションSynageva Biopharma Corp. 組換えヒトnagluタンパク質およびその利用
US9101678B2 (en) 2011-11-03 2015-08-11 Elwha Llc Heat-sanitization of surfaces
US20130121984A1 (en) * 2011-11-07 2013-05-16 Shire Human Genetic Therapies, Inc. Biomarkers for sanfilippo syndrome and uses thereof
AU2012346448B2 (en) * 2011-12-02 2017-09-14 Armagen, Inc. Methods and compositions for increasing arylsulfatase A activity in the CNS
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
EP2793933B1 (en) * 2011-12-23 2019-04-17 Shire Human Genetic Therapies, Inc. Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase
ES2647082T3 (es) * 2012-07-31 2017-12-19 Bioasis Technologies Inc Proteínas desfosforiladas de la enfermedad de depósito lisosómico y métodos de uso de las mismas
WO2014022841A1 (en) * 2012-08-03 2014-02-06 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Cyclodextrin for the treatment of lysosomal storage diseases
HUE039334T2 (hu) 2012-11-27 2018-12-28 Biomarin Pharm Inc Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai
EA201590582A1 (ru) * 2012-12-07 2016-01-29 Шир Хьюман Дженетик Терапис, Инк. Способы и композиции для лечения мукополисахаридоза типа iiia с использованием интратекального введения
EP2981551B1 (en) 2013-02-20 2020-06-03 Valerion Therapeutics, LLC Methods and compositions for treatment of pompe disease
CN105377039A (zh) * 2013-05-15 2016-03-02 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
US9994641B2 (en) 2013-12-25 2018-06-12 Jcr Pharmaceuticals Co., Ltd. Anti-human transferrin receptor antibody that passes through blood-brain barrier
TWI752907B (zh) * 2015-05-08 2022-01-21 美商拜奧馬林製藥公司 用於治療cln2疾病之tpp1調配物及方法
CA2988529A1 (en) 2015-06-10 2016-12-15 Vtesse, Inc. Hydroxypropyl beta-cyclodextrin compositions and methods
US11130815B2 (en) 2015-06-24 2021-09-28 Jcr Pharmaceuticals Co., Ltd. Fusion proteins containing a BDNF and an anti-human transferrin receptor antibody
TWI769982B (zh) 2015-06-24 2022-07-11 日商Jcr製藥股份有限公司 通過血腦障壁之抗人類運鐵蛋白受體抗體
AU2016349528A1 (en) 2015-11-06 2018-05-17 Biomarin Pharmaceutical Inc. Cell-based assays for detection of antibodies or other factors that neutralize uptake of lysosomal enzymes
EP4275751A3 (en) 2016-02-24 2023-12-27 BioMarin Pharmaceutical Inc. Improved naglu fusion protein formulation
IL305449B2 (en) 2016-04-15 2026-01-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous type ii diabetes
KR102657707B1 (ko) * 2016-06-02 2024-04-15 에이디에스 테라퓨틱스 엘엘씨 녹내장 수술 성공을 개선하기 위해 닌테다닙을 사용하는 조성물 및 방법
RU2764919C2 (ru) 2016-06-13 2022-01-24 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Оптимизированные гены и экспрессионные кассеты cln1, и их применение
AU2017385274B2 (en) 2016-12-26 2024-02-22 Jcr Pharmaceuticals Co., Ltd. Fusion protein including BDNF
TWI761413B (zh) 2016-12-26 2022-04-21 日商Jcr製藥股份有限公司 通過血腦障壁之新穎抗人類運鐵蛋白受體抗體
US11723865B2 (en) 2017-02-08 2023-08-15 New Hope Research Foundation, Inc. Systems and methods for enhanced distribution of a biologic agent within the brain and spinal cord
US11541005B2 (en) 2017-02-08 2023-01-03 New Hope Research Foundation, Inc. Systems and methods for enhanced distribution of a biologic agent within the brain and spinal cord
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
AU2018345303B2 (en) 2017-10-02 2025-09-18 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
US11730767B2 (en) 2018-02-23 2023-08-22 Regeneration Biomedical, Inc. Methods, apparatuses and systems for instilling stem cells and pharmaceuticals into the human ventricular system
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2020061366A1 (en) 2018-09-19 2020-03-26 Regeneration Biomedical, Inc. Wnt-activated adipose-derived stem cell apparatuses, methods and systems
EP3670652A1 (en) * 2018-12-18 2020-06-24 NEUWAY Pharma GmbH Vlp for the treatment of a lysosomal storage disease
ES2933275T3 (es) * 2018-12-18 2023-02-03 Neuway Pharma Gmbh Un sistema de suministro de fármacos novedoso y métodos para proporcionar los mismos
CN113301925A (zh) 2018-12-19 2021-08-24 小利兰·斯坦福大学理事会 用于溶酶体靶向的双官能分子以及相关的组合物和方法
US11446084B2 (en) 2019-07-12 2022-09-20 Neuralink Corp. Laser drilling of pia mater
AU2020336984B2 (en) 2019-08-29 2026-04-23 Biomarin Pharmaceutical Inc. Methods for treating CLN2 disease in pediatric subjects
AU2021217816A1 (en) 2020-02-03 2022-08-11 Gt Gain Therapeutics Sa Combination therapy for treating MPS1
KR102880902B1 (ko) * 2021-01-29 2025-11-03 현대자동차주식회사 셀 기반 모빌리티 생산시스템의 운영방법
CA3207482A1 (en) * 2021-02-09 2022-08-18 Jill Wood Nootropic peptides for treating lysosomal storage diseases
AU2022310356A1 (en) 2021-07-14 2024-01-25 Lycia Therapeutics, Inc. Asgpr cell surface receptor binding compounds and conjugates
WO2023156966A1 (en) 2022-02-18 2023-08-24 Beren Therapeutics P.B.C. Compositions of hydroxypropyl-beta-cyclodextrin and methods of purifying the same
WO2025125542A1 (en) * 2023-12-15 2025-06-19 F. Hoffmann-La Roche Ag Intrathecal dosage method

Family Cites Families (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US2601215A (en) 1948-03-17 1952-06-17 American Cyanamid Co Process of preparing dihydropterins
US3505329A (en) 1968-02-06 1970-04-07 Smithkline Corp Process for the synthesis of biopterin
JPS5883691A (ja) 1981-11-13 1983-05-19 Kanegafuchi Chem Ind Co Ltd 1′,2′−ジアシル−(6r,s)−5,6,7,8−テトラヒドロ−l−ビオプテリンおよびその製法
JPS5925323A (ja) 1982-03-03 1984-02-09 鐘淵化学工業株式会社 プテリン誘導体からなる脳内神経伝達物質の関わる疾病の治療剤
CH651755A5 (en) 1982-03-03 1985-10-15 Kanegafuchi Chemical Ind Use of pterin derivatives
JPS5976086A (ja) 1982-09-20 1984-04-28 ザ ウエルカム フアウンデ−シヨン リミテツド プテリジン化合物
GB8318833D0 (en) 1983-07-12 1983-08-10 Wellcome Found Chemical compounds
US5196533A (en) 1983-04-11 1993-03-23 South Alabama Medical Science Foundation, Usa Cyclization of 5 amino-pyrimidines to quinoid 6,6 disubstituted dihydropteridines
US4587340A (en) 1983-09-19 1986-05-06 Burroughs Wellcome Co. Biopterin analogs
JPS60178887A (ja) 1984-02-23 1985-09-12 Kanegafuchi Chem Ind Co Ltd 5,6,7,8−テトラヒドロ−l−ビオプテリンの製造法
JPS60199889A (ja) 1984-03-24 1985-10-09 Kanegafuchi Chem Ind Co Ltd 5,6,7,8−テトラヒドロ−l−エリスロ−ビオプテリンの硫酸塩およびその製法
US4550109A (en) 1984-05-31 1985-10-29 The Board Of Regents, The University Of Texas System Lipoidal biopterin compounds
DE3437944A1 (de) 1984-10-17 1986-07-31 Biotest-Serum-Institut Gmbh, 6000 Frankfurt Verwendung von pterinen zur steigerung der aktivitaet von lymphokinen und anderen blutfaktoren, sowie ein diagnostisches oder therapeutisches praeparat, das pterine in kombination mit lymphokinen enthaelt
US4713454A (en) 1985-01-28 1987-12-15 Shiratori Pharmaceutical Co., Ltd. Preparation process of (6R)-tetrahydro-L-biopterin
JPS61277618A (ja) 1985-06-04 1986-12-08 Suntory Ltd 自閉症治療剤
US4801575A (en) * 1986-07-30 1989-01-31 The Regents Of The University Of California Chimeric peptides for neuropeptide delivery through the blood-brain barrier
US4937342A (en) 1987-11-30 1990-06-26 Kabushiki Kaisha Vitamin Kenkyusyo Intermediates for synthesizing BH4 and its derivatives
US6149909A (en) 1995-06-23 2000-11-21 Women's And Children's Hospital Synthetic α-L-iduronidase and genetic sequences encoding same
JP2534423B2 (ja) 1991-12-26 1996-09-18 コーネル・リサーチ・ファンデーション・インコーポレイテッド 酸化窒素の過剰生産から生じる血管失調を阻止する阻害剤
US5198547A (en) 1992-03-16 1993-03-30 South Alabama Medical Science Foundation, Usa Process for N5-formylating tetrahydropteridines
US5981194A (en) 1992-07-10 1999-11-09 University Of British Columbia Use of p97 and iron binding proteins as diagnostic and therapeutic agents
DE4308739C1 (de) 1993-03-19 1994-06-23 Henning Berlin Gmbh Pterinderivate, ihre Herstellung und ihre Verwendung
US5945452A (en) 1993-06-11 1999-08-31 The Board Of Trustees Of The Leland Stanford Junior University Treatment of vascular degenerative diseases by modulation of endogenous nitric oxide production or activity
US5502050A (en) 1993-11-29 1996-03-26 Cornell Research Foundation, Inc. Blocking utilization of tetrahydrobiopterin to block induction of nitric oxide synthesis
ATE218345T1 (de) 1994-08-05 2002-06-15 Suntory Ltd Arzneimittel gegen spinocerebellare degeneration
US5716614A (en) * 1994-08-05 1998-02-10 Molecular/Structural Biotechnologies, Inc. Method for delivering active agents to mammalian brains in a complex with eicosapentaenoic acid or docosahexaenoic acid-conjugated polycationic carrier
DE69637875D1 (de) 1995-09-14 2009-04-30 Virginia Tech Intell Prop Produktion von lysosomalen enzymen in pflanzlichen expressionssystemen
US6002067A (en) 1996-05-20 1999-12-14 The University Of British Columbia Transgenic mouse model for iduronidase deficiency and methods of making and using same
US20020076782A1 (en) 1996-07-05 2002-06-20 John P.N. Rosazza Purified nitric oxide synthase
GB9617990D0 (en) 1996-08-29 1996-10-09 Scotia Holdings Plc Treatment of pain
US6458574B1 (en) * 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
US5922713A (en) 1997-06-26 1999-07-13 Werner; Ernst Inhibition of nitric oxide synthase
JP4306825B2 (ja) 1998-02-27 2009-08-05 アスビオファーマ株式会社 インスリン抵抗性が関与する血管機能異常を伴う疾患の予防または治療剤
CA2328518A1 (en) * 1998-05-13 1999-11-18 Harbor-Ucla Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US6200758B1 (en) 1999-02-19 2001-03-13 New York State Office Of Mental Health Phenylalanine hydroxylase gene variants, and amino acid and pterin homeostasis, in the definition, detection, treatment and prevention of psychotic, mood and personality disorders
US6217552B1 (en) 1999-03-01 2001-04-17 Coaxia, Inc. Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma
US6521212B1 (en) 1999-03-18 2003-02-18 United Therapeutics Corporation Method for treating peripheral vascular disease by administering benzindene prostaglandins by inhalation
EP1175210A4 (en) 1999-03-19 2003-07-09 Enos Pharmaceuticals Inc STRENGTHENING THE BIODAVAILABILITY OF DRUGS IN THE BRAIN
US6537785B1 (en) 1999-09-14 2003-03-25 Genzyme Glycobiology Research Institute, Inc. Methods of treating lysosomal storage diseases
GB2355191A (en) 1999-10-12 2001-04-18 Laxdale Ltd Combination formulations for fatigue, head injury and strokes
US6569661B1 (en) 1999-11-12 2003-05-27 Biomarin Pharmaceutical Inc. Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
EP1955703A1 (en) * 1999-11-12 2008-08-13 Oncolytics Biotech Inc. Viruses for the treatment of cellular proliferative disorders
US6426208B1 (en) 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US6585971B1 (en) 1999-11-12 2003-07-01 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof
AU1775901A (en) 1999-11-17 2001-05-30 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
US6972124B2 (en) * 2000-05-01 2005-12-06 Biomarin Pharmaceuticals Inc. Precursor of N-acetylgalactosamine-4-sulfatase, methods of treatment using said enzyme and methods for producing and purifying said enzyme
US20020006400A1 (en) 2000-05-11 2002-01-17 Peter Lobel Recombinant human CLN2 protein and methods of its production and use
US6544994B2 (en) 2000-06-07 2003-04-08 Eprov Ag Pharmaceutical preparation for treating or preventing cardiovascular or neurological disorders by modulating of the activity of nitric oxide synthase
WO2001097829A2 (en) * 2000-06-19 2001-12-27 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
AU7194101A (en) 2000-07-18 2002-01-30 Univ Duke Treatment of glycogen storage disease type ii
US20050119270A1 (en) 2000-08-04 2005-06-02 Mason R. P. Synergistic effect of amlodipine and atorvastatin on aortic endothelial cell nitric oxide release
KR100916163B1 (ko) 2000-08-31 2009-09-08 아스비오파마 가부시키가이샤 비오프테린류의 제조방법
ATE384736T1 (de) * 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
AU2002317700A1 (en) 2001-06-07 2002-12-16 Hemebiotech A/S Production of recombinant human arylsulfatase a
US20030007961A1 (en) 2001-06-22 2003-01-09 Wilburn Michael D. Orthomolecular vitamin E derivatives
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
AU2003224880A1 (en) 2002-04-05 2003-10-27 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy
US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
EP1503788B1 (en) 2002-04-25 2011-06-29 Shire Human Genetic Therapies, Inc. Treatment of alpha-galactosidase a deficiency
US7485314B2 (en) 2002-05-06 2009-02-03 Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center Induction of antigen specific immunologic tolerance
US20050142141A1 (en) 2002-11-27 2005-06-30 Pardridge William M. Delivery of enzymes to the brain
DE10260263A1 (de) 2002-12-20 2004-07-15 Biocrates Life Sciences Gmbh Verwendung von Tetrahydrobiopterinderivaten zur Behandlung und Ernährung von Patienten mit Aminosäurestoffwechselstörungen
EP1613264A4 (en) 2003-04-16 2007-08-15 Yeda Res & Dev MEDICAMENT FOR THE TREATMENT OF GAUCHER DISEASE AND METHOD FOR IDENTIFYING IT
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
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
WO2006055511A2 (en) 2004-11-17 2006-05-26 Biomarin Pharmaceutical Inc. Stable tablet formulation of tetrahydrobiopterin

Similar Documents

Publication Publication Date Title
JP2007504166A5 (https=)
US12409210B2 (en) CNS delivery of therapeutic agents
Ellison et al. Advances in therapies for neurological lysosomal storage disorders
US10898551B2 (en) Soluble hyaluronidase glycoprotein (sHASEGP), process for preparing the same, uses and pharmaceutical compositions comprising thereof
Chen et al. Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future
JP2013534526A5 (https=)
EP1981546B1 (en) Enzyme replacement therapy for treating lysosomal storage diseases
JP5819574B2 (ja) 脳およびその他の組織への治療用化合物の送達
Kakkis et al. Enzyme replacement therapy in feline mucopolysaccharidosis I
JP2016037504A5 (https=)
Ortolano et al. Treatment of lysosomal storage diseases: recent patents and future strategies
CA2803166A1 (en) Cns delivery of therapeutic agents
JP2015524662A5 (https=)
Abasolo et al. Nanotechnology‐based approaches for treating lysosomal storage disorders, a focus on Fabry disease
Safary et al. Targeted enzyme delivery systems in lysosomal disorders: an innovative form of therapy for mucopolysaccharidosis
US10138474B2 (en) Scavenger receptor uptake for fabry disease enzyme replacement therapy
US20180326021A1 (en) Methods and Compositions for the Treatment of Cytoplasmic Glycogen Storage Disorders
WO2017049161A1 (en) ACID ALPHA-GLUCOSIDASE AND A β-2 AGONIST FOR THE TREATMENT OF LYSOSOMAL STORAGE DISORDER
Matzner Therapy of lysosomal storage diseases
WO2019145500A1 (en) Method of treatment
Zia et al. Therapy for neuropathic lysosomal storage diseases
HK40059834A (en) Pharmaceutical formulation comprising a replacement enzyme for a lysosomal enzyme for use in treating lysosomal storage disease intrathecally
HK40126595A (zh) 用於预防和治疗患有黏多糖贮积症ii型的受试者的溶酶体酶缺乏的药物组合物和方法
HK1230954B (en) Cns delivery of therapeutic agents
HK1230954A1 (en) Cns delivery of therapeutic agents