DK2666476T3 - Intraventrikulær enzymadministration ved lysosomale aflejringssygdomme - Google Patents
Intraventrikulær enzymadministration ved lysosomale aflejringssygdomme Download PDFInfo
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- DK2666476T3 DK2666476T3 DK13173895.7T DK13173895T DK2666476T3 DK 2666476 T3 DK2666476 T3 DK 2666476T3 DK 13173895 T DK13173895 T DK 13173895T DK 2666476 T3 DK2666476 T3 DK 2666476T3
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Classifications
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- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
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- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
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- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- A—HUMAN NECESSITIES
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- A61P13/00—Drugs for disorders of the urinary system
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- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C—CHEMISTRY; METALLURGY
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- C12Y—ENZYMES
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- C12Y301/04—Phosphoric diester hydrolases (3.1.4)
- C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
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- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
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- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
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- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Dermatology (AREA)
- Obesity (AREA)
- Pulmonology (AREA)
- Urology & Nephrology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Infusion, Injection, And Reservoir Apparatuses (AREA)
Claims (5)
1. Syre-sphingomyelinase til anvendelse til forebyggelse eller behandling af Niemann-Picks sygdom type A eller B hos en patient, hvor forebyggelsen eller behandlingen omfatter intraventrikulær administration af syre-sphingomyelinasen til patientens hjerne, hvor administrationen af en enkelt dosis af syre-sphingomyelinasen varer mere end 5 timer.
2. Syre-sphingomyelinase til anvendelse ifølge krav 1, hvor forebyggelsen eller behandlingen omfatter administration af syre-sphingomyelinasen til de laterale ventrikler og/eller den fjerde ventrikel i hjernen.
3. Syre-sphingomyelinase til anvendelse ifølge krav 1 eller krav 2, hvor forebyggelsen eller behandlingen omfatter bestemmelse af reakkumuleringen af sphingomyelin i hjernen og/eller i et eller flere af de viscerale organer.
4. Syre-sphingomyelinase til anvendelse ifølge et hvilket som helst af kravene 1 til 3, hvor syre-sphingomyelinasen skal administreres ved anvendelse af en pumpe og/eller et kateter.
5. Syre-sphingomyelinase til anvendelse ifølge et hvilket som helst af kravene 1 til 4, hvor administrationen omfatter en flerhed af infusioner af syre-sphingomyelinase.
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| US76037806P | 2006-01-20 | 2006-01-20 | |
| EP07716858.1A EP1984018B1 (en) | 2006-01-20 | 2007-01-22 | Intraventricular enzyme delivery for lysosomal storage diseases |
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| DK07716858.1T DK1984018T3 (da) | 2006-01-20 | 2007-01-22 | Intraventrikulær enzymindgivelse ved lysosomale lagringssygdomme |
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| AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| HUE040490T2 (hu) * | 2006-02-09 | 2019-03-28 | Genzyme Corp | Lassú intraventrikuláris szállítás |
| EP2154969B1 (en) | 2007-05-16 | 2015-11-18 | The Brigham and Women's Hospital, Inc. | Treatment of synucleinopathies |
| CN102369023A (zh) * | 2008-12-16 | 2012-03-07 | 建新公司 | 寡糖-蛋白缀合物 |
| HRP20250897T1 (hr) | 2009-05-02 | 2025-09-26 | Genzyme Corporation | Genska terapija za neurodegenerativne poremećaje |
| SI3998078T1 (sl) * | 2009-08-28 | 2026-02-27 | Genzyme Corporation | Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze |
| FI3205351T3 (fi) | 2010-04-23 | 2023-07-06 | Alexion Pharma Inc | Lysosomaalisen kertymätaudin entsyymi |
| US20110318324A1 (en) * | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of b-galactocerebrosidase |
| KR20230159646A (ko) | 2010-06-25 | 2023-11-21 | 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 |
| PT3103469T (pt) | 2010-06-25 | 2021-03-04 | Shire Human Genetic Therapies | Administração de agentes terapêuticos no snc |
| LT2588130T (lt) * | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas cns |
| HRP20191923T1 (hr) * | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku arilsulfataze a u cns |
| JP2012062312A (ja) * | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | ハンター症候群の治療剤 |
| KR20150038636A (ko) | 2010-09-09 | 2015-04-08 | 시나게바 바이오파르마, 코포레이션 | 환자에서 리소좀 산 리파제 결핍증의 치료를 위한 리소좀 산 리파제의 용도 |
| US9387236B2 (en) * | 2011-06-10 | 2016-07-12 | Prothera Inc. | Pharmaceutical compositions containing protease and methods for the treatment of lysosomal storage diseases |
| US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| 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 |
| EA039246B1 (ru) * | 2012-12-06 | 2021-12-22 | Шир Хьюман Дженетик Терапис, Инк. | Способ лечения когнитивных нарушений, связанных с синдромом хантера |
| AU2014218854B2 (en) | 2013-02-20 | 2019-01-24 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Pompe disease |
| ES2762608T3 (es) | 2013-06-07 | 2020-05-25 | Genzyme Corp | Marcador para trastornos de esfingomielinasa ácida y usos del mismo |
| EP3088518A4 (en) | 2013-12-25 | 2017-09-20 | JCR Pharmaceuticals Co., Ltd. | Novel anti-transferrin receptor antibody that passes through blood-brain barrier |
| US10357542B2 (en) | 2014-02-04 | 2019-07-23 | New York University | Progranulin (PGRN) and its derivatives for diagnosis and treatment of lysosomal storage diseases |
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| EA039366B1 (ru) | 2015-06-24 | 2022-01-19 | Джей Си Ар ФАРМАСЬЮТИКАЛЗ КО., ЛТД. | Антитело к рецептору трансферрина человека, проникающее через гематоэнцефалический барьер |
| WO2017024137A1 (en) * | 2015-08-04 | 2017-02-09 | New York University | Progranulin (pgrn) fragments and derivatives for treatment or alleviation of lysosomal storage diseases |
| RU2634113C1 (ru) * | 2016-11-18 | 2017-10-24 | Государственное бюджетное учреждение "Академия наук Республики Саха (Якутия)" (ГБУ АН РС(Я)) | Способ фиксации головного мозга ископаемого позднеплейстоценового млекопитающего |
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- 2007-01-19 AR ARP070100239A patent/AR059089A1/es not_active Application Discontinuation
- 2007-01-22 TR TR2019/03855T patent/TR201903855T4/tr unknown
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- 2007-01-22 RU RU2008134120/15A patent/RU2529830C2/ru not_active IP Right Cessation
- 2007-01-22 EP EP18212320.8A patent/EP3517124A1/en not_active Withdrawn
- 2007-01-22 JP JP2008551442A patent/JP5681345B2/ja not_active Expired - Fee Related
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- 2007-01-22 CA CA2966155A patent/CA2966155A1/en not_active Abandoned
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