CR20150638A - Marcador para trastornos de la esfingomielinasa ácida y sus usos - Google Patents

Marcador para trastornos de la esfingomielinasa ácida y sus usos

Info

Publication number
CR20150638A
CR20150638A CR20150638A CR20150638A CR20150638A CR 20150638 A CR20150638 A CR 20150638A CR 20150638 A CR20150638 A CR 20150638A CR 20150638 A CR20150638 A CR 20150638A CR 20150638 A CR20150638 A CR 20150638A
Authority
CR
Costa Rica
Prior art keywords
disorders
acid sphingomyelinase
marker
asm
treatment
Prior art date
Application number
CR20150638A
Other languages
English (en)
Inventor
Wei-Lien Chuang
Gerald F Cox
X Kate Zhang
Original Assignee
Genzyme Corp
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of CR20150638A publication Critical patent/CR20150638A/es

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/92Chemical 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/04Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04012Sphingomyelin phosphodiesterase (3.1.4.12)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90Enzymes; Proenzymes
    • G01N2333/914Hydrolases (3)
    • G01N2333/916Hydrolases (3) acting on ester bonds (3.1), e.g. phosphatases (3.1.3), phospholipases C or phospholipases D (3.1.4)
    • G01N2333/922Ribonucleases (RNAses); Deoxyribonucleases (DNAses)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2405/00Assays, e.g. immunoassays or enzyme assays, involving lipids
    • G01N2405/08Sphingolipids
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/04Endocrine or metabolic disorders
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/52Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

Landscapes

  • 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)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Biomedical Technology (AREA)
  • Urology & Nephrology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Pathology (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • General Physics & Mathematics (AREA)
  • Food Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Endocrinology (AREA)
  • Pulmonology (AREA)
  • Diabetes (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)

Abstract

La presente descripción proporciona métodos de evaluación, diagnóstico, control y/o tratamiento de trastornos de la esfingomielinasa ácida (ASM), tales corno la enfermedad deNiemann-Pick. En particular, los métodos abarcan técnicas paradiagnóstico y/o tratamiento mejorados de un trastorno de la ASM,por ejemplo usando terapia de sustitución de enzimas.
CR20150638A 2013-06-07 2015-12-04 Marcador para trastornos de la esfingomielinasa ácida y sus usos CR20150638A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361832302P 2013-06-07 2013-06-07
PCT/US2014/041405 WO2014197859A1 (en) 2013-06-07 2014-06-06 Marker for acid sphingomyelinase disorders and uses thereof

Publications (1)

Publication Number Publication Date
CR20150638A true CR20150638A (es) 2016-02-08

Family

ID=51062988

Family Applications (1)

Application Number Title Priority Date Filing Date
CR20150638A CR20150638A (es) 2013-06-07 2015-12-04 Marcador para trastornos de la esfingomielinasa ácida y sus usos

Country Status (31)

Country Link
US (3) US10022428B2 (es)
EP (2) EP3584580A1 (es)
JP (4) JP6457501B2 (es)
KR (3) KR102228367B1 (es)
CN (2) CN105474017A (es)
AU (4) AU2014274670B2 (es)
BR (1) BR112015030099A2 (es)
CA (1) CA2914751C (es)
CL (1) CL2015003563A1 (es)
CR (1) CR20150638A (es)
DK (1) DK3004896T3 (es)
EA (1) EA035342B1 (es)
ES (1) ES2762608T3 (es)
HK (1) HK1217534A1 (es)
HR (1) HRP20192246T1 (es)
HU (1) HUE047863T2 (es)
IL (3) IL284102B2 (es)
LT (1) LT3004896T (es)
MA (1) MA38638B1 (es)
MX (1) MX370570B (es)
MY (1) MY185990A (es)
NZ (1) NZ754328A (es)
PH (1) PH12015502687A1 (es)
PL (1) PL3004896T3 (es)
PT (1) PT3004896T (es)
RS (1) RS59677B1 (es)
SG (2) SG10201709925SA (es)
SI (1) SI3004896T1 (es)
UA (1) UA120591C2 (es)
WO (1) WO2014197859A1 (es)
ZA (1) ZA201508630B (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
LT3004896T (lt) * 2013-06-07 2020-01-10 Genzyme Corporation Rūgštinės sfingomielinazės sutrikimų žymuo ir jo panaudojimas
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
PL3672622T3 (pl) * 2017-08-24 2024-04-02 Genzyme Corporation Rekombinowana ludzka kwaśna sfingomielinaza do zastosowania w leczeniu nieprawidłowych stanów kości u pacjentów z niedoborem kwaśnej sfingomielinazy
MX2020002918A (es) 2017-10-02 2020-07-22 Denali Therapeutics Inc Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas.
WO2024026390A2 (en) * 2022-07-28 2024-02-01 Wylder Nation Foundation Compositions and methods for diagnosing, treating, and preventing lysosomal storage diseases

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US4039388A (en) 1976-06-04 1977-08-02 The United States Of America As Represented By The Government Diagnostic test for Niemann-Pick disease
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
JP2009519713A (ja) 2005-12-15 2009-05-21 ザ リサーチ ファウンデーション オブ ステート ユニバーシティ オブ ニューヨーク 血漿中及び組織中のスフィンゴエミリン及びホスファチジルコリンの酵素的測定方法
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
KR102608551B1 (ko) * 2009-08-28 2023-12-04 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
ES2731920T3 (es) * 2013-05-14 2019-11-19 Centogene Ag Método para el diagnóstico de la enfermedad de Niemann-Pick
LT3004896T (lt) * 2013-06-07 2020-01-10 Genzyme Corporation Rūgštinės sfingomielinazės sutrikimų žymuo ir jo panaudojimas

Also Published As

Publication number Publication date
JP2019089767A (ja) 2019-06-13
SI3004896T1 (sl) 2020-01-31
CN105474017A (zh) 2016-04-06
CN115128287A (zh) 2022-09-30
US20210162021A1 (en) 2021-06-03
CA2914751A1 (en) 2014-12-11
KR20210104177A (ko) 2021-08-24
KR102228367B1 (ko) 2021-03-16
RS59677B1 (sr) 2020-01-31
AU2018200977B2 (en) 2020-07-23
LT3004896T (lt) 2020-01-10
AU2020257114A1 (en) 2020-11-19
PH12015502687A1 (en) 2016-03-07
JP2016526050A (ja) 2016-09-01
IL284102A (en) 2021-07-29
CA2914751C (en) 2023-10-03
NZ715001A (en) 2021-10-29
EA035342B1 (ru) 2020-05-29
MA38638A1 (fr) 2017-02-28
JP6835806B2 (ja) 2021-02-24
MX370570B (es) 2019-12-17
ES2762608T3 (es) 2020-05-25
SG11201509622WA (en) 2015-12-30
US20160120958A1 (en) 2016-05-05
DK3004896T3 (da) 2020-01-06
MY185990A (en) 2021-06-14
IL284102B (en) 2022-12-01
IL242964B (en) 2021-07-29
AU2018200977A1 (en) 2018-03-01
WO2014197859A1 (en) 2014-12-11
EA201592265A1 (ru) 2016-04-29
CL2015003563A1 (es) 2016-08-05
HRP20192246T1 (hr) 2020-03-06
HK1217534A1 (zh) 2017-01-13
KR20160018651A (ko) 2016-02-17
AU2014274670B2 (en) 2017-11-09
KR102292298B1 (ko) 2021-08-24
US10888607B2 (en) 2021-01-12
NZ754328A (en) 2021-12-24
JP6457501B2 (ja) 2019-01-23
BR112015030099A2 (pt) 2017-07-25
KR20210031541A (ko) 2021-03-19
AU2014274670A1 (en) 2016-01-07
EP3004896B1 (en) 2019-09-18
US10022428B2 (en) 2018-07-17
EP3004896A1 (en) 2016-04-13
MA38638B1 (fr) 2018-03-30
IL284102B2 (en) 2023-04-01
JP7414750B2 (ja) 2024-01-16
ZA201508630B (en) 2020-01-29
PT3004896T (pt) 2019-12-23
HUE047863T2 (hu) 2020-05-28
UA120591C2 (uk) 2020-01-10
IL298675A (en) 2023-01-01
US20180289778A1 (en) 2018-10-11
EP3584580A1 (en) 2019-12-25
AU2020257114B2 (en) 2023-03-16
JP2024038145A (ja) 2024-03-19
PL3004896T3 (pl) 2020-04-30
SG10201709925SA (en) 2017-12-28
US11998592B2 (en) 2024-06-04
JP2021075544A (ja) 2021-05-20
KR102434244B1 (ko) 2022-08-19
MX2015016844A (es) 2016-04-04
AU2023201062A1 (en) 2023-03-23

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