JP6457501B2 - 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用 - Google Patents

酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用 Download PDF

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JP6457501B2
JP6457501B2 JP2016518053A JP2016518053A JP6457501B2 JP 6457501 B2 JP6457501 B2 JP 6457501B2 JP 2016518053 A JP2016518053 A JP 2016518053A JP 2016518053 A JP2016518053 A JP 2016518053A JP 6457501 B2 JP6457501 B2 JP 6457501B2
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JP2016526050A5 (enExample
JP2016526050A (ja
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ウエイ−リエン・チュワーン
ジェラルド・エフ・コックス
エックス・ケイト・ジャーン
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • 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
    • 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)
    • C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • 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)
    • G01N2333/922—Ribonucleases (RNAses); Deoxyribonucleases (DNAses)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2405/00—Assays, e.g. immunoassays or enzyme assays, involving lipids
    • G01N2405/08—Sphingolipids
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/04—Endocrine or metabolic disorders
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Analytical Chemistry (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Biotechnology (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Endocrinology (AREA)
  • Food Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Pulmonology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
JP2016518053A 2013-06-07 2014-06-06 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用 Active JP6457501B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Related Child Applications (1)

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JP2018236856A Division JP6835806B2 (ja) 2013-06-07 2018-12-19 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用

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JP2016526050A JP2016526050A (ja) 2016-09-01
JP2016526050A5 JP2016526050A5 (enExample) 2017-07-13
JP6457501B2 true JP6457501B2 (ja) 2019-01-23

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JP2016518053A Active JP6457501B2 (ja) 2013-06-07 2014-06-06 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用
JP2018236856A Active JP6835806B2 (ja) 2013-06-07 2018-12-19 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用
JP2021015390A Active JP7414750B2 (ja) 2013-06-07 2021-02-03 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用
JP2023220385A Pending JP2024038145A (ja) 2013-06-07 2023-12-27 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用

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JP2021015390A Active JP7414750B2 (ja) 2013-06-07 2021-02-03 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用
JP2023220385A Pending JP2024038145A (ja) 2013-06-07 2023-12-27 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用

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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019089767A (ja) * 2013-06-07 2019-06-13 ジェンザイム・コーポレーション 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102094253B1 (ko) * 2009-08-28 2020-03-31 이칸 스쿨 오브 메디슨 엣 마운트 시나이 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
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
SG11202001544VA (en) * 2017-08-24 2020-03-30 Sanofi Sa Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients
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
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
EP4562432A2 (en) * 2022-07-28 2025-06-04 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
CA2634042A1 (en) 2005-12-15 2007-07-12 The Research Foundation Of State University Of New York Enzymatic methods for measuring plasma and tissue sphingomylelin and phosphatidylcholine
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
KR102094253B1 (ko) * 2009-08-28 2020-03-31 이칸 스쿨 오브 메디슨 엣 마운트 시나이 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
JP2013002933A (ja) 2011-06-16 2013-01-07 Hitachi Plant Technologies Ltd ３次元測位システム
JP6243846B2 (ja) * 2011-11-15 2017-12-06 セントジーン アーゲー ニーマン・ピック病の診断のための方法
WO2014183873A1 (en) * 2013-05-14 2014-11-20 Centogene Ag Method for the diagnosis of niemann-pick disease
PL3004896T3 (pl) * 2013-06-07 2020-04-30 Genzyme Corporation Marker zaburzeń kwaśnej spfingomielinazy i jego zastosowania

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019089767A (ja) * 2013-06-07 2019-06-13 ジェンザイム・コーポレーション 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用

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BR112015030099A2 (pt) 2017-07-25
AU2014274670B2 (en) 2017-11-09
US20240398907A1 (en) 2024-12-05
AU2023201062A1 (en) 2023-03-23
EP3584580A1 (en) 2019-12-25
WO2014197859A1 (en) 2014-12-11
CR20150638A (es) 2016-02-08
KR20210031541A (ko) 2021-03-19
MX370570B (es) 2019-12-17
PH12015502687A1 (en) 2016-03-07
HRP20192246T1 (hr) 2020-03-06
IL284102B (en) 2022-12-01
JP7414750B2 (ja) 2024-01-16
EA201592265A1 (ru) 2016-04-29
KR102292298B1 (ko) 2021-08-24
SG10201709925SA (en) 2017-12-28
AU2014274670A1 (en) 2016-01-07
US10888607B2 (en) 2021-01-12
CL2015003563A1 (es) 2016-08-05
MA38638A1 (fr) 2017-02-28
AU2018200977A1 (en) 2018-03-01
DK3004896T3 (da) 2020-01-06
KR20210104177A (ko) 2021-08-24
KR102434244B1 (ko) 2022-08-19
IL284102A (en) 2021-07-29
US11998592B2 (en) 2024-06-04
JP2016526050A (ja) 2016-09-01
US20180289778A1 (en) 2018-10-11
MA38638B1 (fr) 2018-03-30
IL298675B2 (en) 2025-12-01
PT3004896T (pt) 2019-12-23
NZ715001A (en) 2021-10-29
KR102228367B1 (ko) 2021-03-16
RS59677B1 (sr) 2020-01-31
EP3004896A1 (en) 2016-04-13
CN105474017A (zh) 2016-04-06
IL298675B1 (en) 2025-08-01
AU2023201062B2 (en) 2025-05-29
CA2914751C (en) 2023-10-03
AU2020257114B2 (en) 2023-03-16
AU2025220876A1 (en) 2025-09-11
PL3004896T3 (pl) 2020-04-30
IL284102B2 (en) 2023-04-01
AU2020257114A1 (en) 2020-11-19
US20160120958A1 (en) 2016-05-05
ZA201508630B (en) 2020-01-29
KR20160018651A (ko) 2016-02-17
HK1217534A1 (en) 2017-01-13
EP3004896B1 (en) 2019-09-18
MY185990A (en) 2021-06-14
JP2024038145A (ja) 2024-03-19
SG11201509622WA (en) 2015-12-30
ES2762608T3 (es) 2020-05-25
US20210162021A1 (en) 2021-06-03
JP2021075544A (ja) 2021-05-20
AU2018200977B2 (en) 2020-07-23
EA035342B1 (ru) 2020-05-29
MX2015016844A (es) 2016-04-04
US10022428B2 (en) 2018-07-17
JP6835806B2 (ja) 2021-02-24
UA120591C2 (uk) 2020-01-10
NZ754328A (en) 2021-12-24
HUE047863T2 (hu) 2020-05-28
IL242964B (en) 2021-07-29
IL298675A (en) 2023-01-01
CN115128287A (zh) 2022-09-30
LT3004896T (lt) 2020-01-10
JP2019089767A (ja) 2019-06-13
SI3004896T1 (sl) 2020-01-31
CA2914751A1 (en) 2014-12-11

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