CN105474017A - 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 PDF

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Publication number
CN105474017A
CN105474017A CN201480043918.2A CN201480043918A CN105474017A CN 105474017 A CN105474017 A CN 105474017A CN 201480043918 A CN201480043918 A CN 201480043918A CN 105474017 A CN105474017 A CN 105474017A
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China
Prior art keywords
level
spm
haemolysis
asm
sample
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Pending
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CN201480043918.2A
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English (en)
Chinese (zh)
Inventor
W-L·庄
G·F·考克斯
X·K·张
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Genzyme Corp
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Genzyme Corp
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Priority to CN202210555370.6A priority Critical patent/CN115128287A/zh
Publication of CN105474017A publication Critical patent/CN105474017A/zh
Pending legal-status Critical Current

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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)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
CN201480043918.2A 2013-06-07 2014-06-06 酸性鞘磷脂酶病症的标志物及其用途 Pending CN105474017A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202210555370.6A CN115128287A (zh) 2013-06-07 2014-06-06 酸性鞘磷脂酶病症的标志物及其用途

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)

Application Number Title Priority Date Filing Date
CN202210555370.6A Division CN115128287A (zh) 2013-06-07 2014-06-06 酸性鞘磷脂酶病症的标志物及其用途

Publications (1)

Publication Number Publication Date
CN105474017A true CN105474017A (zh) 2016-04-06

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CN201480043918.2A Pending CN105474017A (zh) 2013-06-07 2014-06-06 酸性鞘磷脂酶病症的标志物及其用途
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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EP (2) EP3584580A1 (enExample)
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CN (2) CN105474017A (enExample)
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CA (1) CA2914751C (enExample)
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PL (1) PL3004896T3 (enExample)
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RS (1) RS59677B1 (enExample)
SG (2) SG11201509622WA (enExample)
SI (1) SI3004896T1 (enExample)
UA (1) UA120591C2 (enExample)
WO (1) WO2014197859A1 (enExample)
ZA (1) ZA201508630B (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
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CN111344003A (zh) * 2017-08-24 2020-06-26 赛诺菲 酸性鞘磷脂酶缺乏症患者中异常骨病症的治疗

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BR112012004377A2 (pt) * 2009-08-28 2017-12-12 Genzyme Corp terapia de reposição enzimática com progressão de dose para tratamento de deficiência de esfingomielinase ácida.
KR102228367B1 (ko) * 2013-06-07 2021-03-16 젠자임 코포레이션 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도
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
CA3076369A1 (en) 2017-10-02 2019-04-11 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
HUE071194T2 (hu) * 2019-05-31 2025-08-28 Genzyme Corp Kétdimenziós LC-MS/MS rendszerek
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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CN102596232A (zh) * 2009-08-28 2012-07-18 西奈山医学院 用于治疗酸性鞘磷脂酶缺乏的剂量递增酶替代疗法
WO2013072060A2 (en) * 2011-11-15 2013-05-23 Centogene Gmbh Method for the diagnosis of niemann-pick disease

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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
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US7615224B2 (en) 2004-12-02 2009-11-10 Women's And Children's Hospital Multiplex-bead complex for determination of lysosomal storage disorders
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CN102596232A (zh) * 2009-08-28 2012-07-18 西奈山医学院 用于治疗酸性鞘磷脂酶缺乏的剂量递增酶替代疗法
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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111344003A (zh) * 2017-08-24 2020-06-26 赛诺菲 酸性鞘磷脂酶缺乏症患者中异常骨病症的治疗
CN111344003B (zh) * 2017-08-24 2024-05-07 建新公司 酸性鞘磷脂酶缺乏症患者中异常骨病症的治疗

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KR102292298B1 (ko) 2021-08-24
MA38638B1 (fr) 2018-03-30
JP2019089767A (ja) 2019-06-13
IL284102B (en) 2022-12-01
PH12015502687A1 (en) 2016-03-07
EP3004896A1 (en) 2016-04-13
IL298675B1 (en) 2025-08-01
AU2014274670B2 (en) 2017-11-09
AU2014274670A1 (en) 2016-01-07
KR102228367B1 (ko) 2021-03-16
AU2020257114B2 (en) 2023-03-16
EP3584580A1 (en) 2019-12-25
US11998592B2 (en) 2024-06-04
EP3004896B1 (en) 2019-09-18
US20180289778A1 (en) 2018-10-11
AU2023201062A1 (en) 2023-03-23
MX370570B (es) 2019-12-17
HRP20192246T1 (hr) 2020-03-06
US20160120958A1 (en) 2016-05-05
UA120591C2 (uk) 2020-01-10
KR102434244B1 (ko) 2022-08-19
IL298675B2 (en) 2025-12-01
RS59677B1 (sr) 2020-01-31
DK3004896T3 (da) 2020-01-06
US20210162021A1 (en) 2021-06-03
US20240398907A1 (en) 2024-12-05
NZ754328A (en) 2021-12-24
MX2015016844A (es) 2016-04-04
MA38638A1 (fr) 2017-02-28
LT3004896T (lt) 2020-01-10
SG10201709925SA (en) 2017-12-28
HUE047863T2 (hu) 2020-05-28
WO2014197859A1 (en) 2014-12-11
ZA201508630B (en) 2020-01-29
PT3004896T (pt) 2019-12-23
CA2914751A1 (en) 2014-12-11
CR20150638A (es) 2016-02-08
IL242964B (en) 2021-07-29
PL3004896T3 (pl) 2020-04-30
SI3004896T1 (sl) 2020-01-31
IL298675A (en) 2023-01-01
BR112015030099A2 (pt) 2017-07-25
EA035342B1 (ru) 2020-05-29
IL284102B2 (en) 2023-04-01
KR20210104177A (ko) 2021-08-24
US10022428B2 (en) 2018-07-17
JP2021075544A (ja) 2021-05-20
IL284102A (en) 2021-07-29
JP2024038145A (ja) 2024-03-19
CN115128287A (zh) 2022-09-30
KR20210031541A (ko) 2021-03-19
SG11201509622WA (en) 2015-12-30
AU2018200977B2 (en) 2020-07-23
AU2023201062B2 (en) 2025-05-29
ES2762608T3 (es) 2020-05-25
JP7414750B2 (ja) 2024-01-16
US10888607B2 (en) 2021-01-12
EA201592265A1 (ru) 2016-04-29
CL2015003563A1 (es) 2016-08-05
HK1217534A1 (en) 2017-01-13
MY185990A (en) 2021-06-14
AU2018200977A1 (en) 2018-03-01
JP6835806B2 (ja) 2021-02-24
AU2025220876A1 (en) 2025-09-11
CA2914751C (en) 2023-10-03
NZ715001A (en) 2021-10-29
JP6457501B2 (ja) 2019-01-23
AU2020257114A1 (en) 2020-11-19
JP2016526050A (ja) 2016-09-01
KR20160018651A (ko) 2016-02-17

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