CN115128287A - 酸性鞘磷脂酶病症的标志物及其用途 - Google Patents
酸性鞘磷脂酶病症的标志物及其用途 Download PDFInfo
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- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- 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
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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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- A—HUMAN NECESSITIES
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- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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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)
- C12Y301/04—Phosphoric diester hydrolases (3.1.4)
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- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/90—Enzymes; Proenzymes
- G01N2333/914—Hydrolases (3)
- G01N2333/916—Hydrolases (3) acting on ester bonds (3.1), e.g. phosphatases (3.1.3), phospholipases C or phospholipases D (3.1.4)
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- G—PHYSICS
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- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
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- G—PHYSICS
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- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis
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- G—PHYSICS
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- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/92—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving lipids, e.g. cholesterol, lipoproteins, or their receptors
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- Proteomics, Peptides & Aminoacids (AREA)
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| US201361832302P | 2013-06-07 | 2013-06-07 | |
| US61/832,302 | 2013-06-07 | ||
| PCT/US2014/041405 WO2014197859A1 (en) | 2013-06-07 | 2014-06-06 | Marker for acid sphingomyelinase disorders and uses thereof |
| CN201480043918.2A CN105474017A (zh) | 2013-06-07 | 2014-06-06 | 酸性鞘磷脂酶病症的标志物及其用途 |
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| CN201480043918.2A Division CN105474017A (zh) | 2013-06-07 | 2014-06-06 | 酸性鞘磷脂酶病症的标志物及其用途 |
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| CN115128287A true CN115128287A (zh) | 2022-09-30 |
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| CN202210555370.6A Pending CN115128287A (zh) | 2013-06-07 | 2014-06-06 | 酸性鞘磷脂酶病症的标志物及其用途 |
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| PL2470199T3 (pl) * | 2009-08-28 | 2019-11-29 | Icahn School Med Mount Sinai | Enzymatyczna terapia zastępcza ze zwiększaniem dawki do leczenia niedoboru kwaśnej sfingomielinazy |
| SI3004896T1 (sl) * | 2013-06-07 | 2020-01-31 | Genzyme Corporation | Označevalec za motnje kisle sfingomielinaze in njegova uporaba |
| JP6984854B2 (ja) * | 2015-10-08 | 2021-12-22 | 国立大学法人千葉大学 | ニーマン・ピック病ｃ型を予防または治療するための医薬組成物 |
| US11898175B2 (en) * | 2017-08-24 | 2024-02-13 | Genzyme Corporation | Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients |
| SG11202002730XA (en) | 2017-10-02 | 2020-04-29 | Denali Therapeutics Inc | Fusion proteins comprising enzyme replacement therapy enzymes |
| JP7568648B2 (ja) * | 2019-05-31 | 2024-10-16 | ジェンザイム・コーポレーション | 二次元ｌｃ－ｍｓ／ｍｓシステム |
| WO2024026390A2 (en) * | 2022-07-28 | 2024-02-01 | Wylder Nation Foundation | Compositions and methods for diagnosing, treating, and preventing lysosomal storage diseases |
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| US4039388A (en) | 1976-06-04 | 1977-08-02 | The United States Of America As Represented By The Government | Diagnostic test for Niemann-Pick disease |
| US4082781A (en) | 1976-06-04 | 1978-04-04 | The United States Of America As Represented By The Department Of Health, Education And Welfare | Synthesis of 2-alkanoylamino-4-nitrophenyl phosphorylcholine-hydroxide |
| US5773278A (en) | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
| WO2002018539A2 (en) | 2000-08-25 | 2002-03-07 | Lysometrix Corporation | Method for assaying the activity of lysosomal enzymes |
| US7001994B2 (en) | 2001-01-18 | 2006-02-21 | Genzyme Corporation | Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins |
| US7615224B2 (en) | 2004-12-02 | 2009-11-10 | Women's And Children's Hospital | Multiplex-bead complex for determination of lysosomal storage disorders |
| JP2006214731A (ja) | 2005-02-01 | 2006-08-17 | Institute Of Physical & Chemical Research | スフィンゴミエリンの検出方法 |
| KR20080082984A (ko) | 2005-12-15 | 2008-09-12 | 더 리서치 파운데이션 오브 스테이트 유니버시티 오브 뉴욕 | 혈장 및 조직의 스핑고마이엘린 및 포스파티딜콜린의효소적 측정 방법 |
| AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| US8026099B2 (en) | 2007-07-26 | 2011-09-27 | Washington University | Lipid profile as a biomarker for early detection of neurological disorders |
| US8497122B2 (en) | 2008-04-11 | 2013-07-30 | Washington University | Biomarkers for Niemann-pick C disease and related disorders |
| PL2470199T3 (pl) | 2009-08-28 | 2019-11-29 | Icahn School Med Mount Sinai | Enzymatyczna terapia zastępcza ze zwiększaniem dawki do leczenia niedoboru kwaśnej sfingomielinazy |
| JP2013002933A (ja) | 2011-06-16 | 2013-01-07 | Hitachi Plant Technologies Ltd | ３次元測位システム |
| WO2013072060A2 (en) * | 2011-11-15 | 2013-05-23 | Centogene Gmbh | Method for the diagnosis of niemann-pick disease |
| DK2997384T3 (da) * | 2013-05-14 | 2019-07-22 | Centogene Ag | Fremgangsmåde til diagnose af niemann-pick sygdom |
| SI3004896T1 (sl) * | 2013-06-07 | 2020-01-31 | Genzyme Corporation | Označevalec za motnje kisle sfingomielinaze in njegova uporaba |
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