JP2015504514A - サンフィリポ症候群のためのバイオマーカーおよびそれらの使用 - Google Patents

サンフィリポ症候群のためのバイオマーカーおよびそれらの使用 Download PDF

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JP2015504514A
JP2015504514A JP2014541200A JP2014541200A JP2015504514A JP 2015504514 A JP2015504514 A JP 2015504514A JP 2014541200 A JP2014541200 A JP 2014541200A JP 2014541200 A JP2014541200 A JP 2014541200A JP 2015504514 A JP2015504514 A JP 2015504514A
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biomarkers
level
syndrome
treatment
sample
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JP2015504514A5 (enExample
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パトリック ハスレット,
パトリック ハスレット,
チャーリー リチャード,
チャーリー リチャード,
Original Assignee
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
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
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    • 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)
    • 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
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P17/14Drugs for dermatological disorders for baldness or alopecia
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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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    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/106Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/118Prognosis of disease development
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/158Expression markers
    • 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
    • G01N2333/435Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
    • G01N2333/475Assays involving growth factors
    • G01N2333/4753Hepatocyte growth factor; Scatter factor; Tumor cytotoxic factor II
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2400/00Assays, e.g. immunoassays or enzyme assays, involving carbohydrates
    • G01N2400/10Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2440/00Post-translational modifications [PTMs] in chemical analysis of biological material
    • G01N2440/14Post-translational modifications [PTMs] in chemical analysis of biological material phosphorylation
    • 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
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    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/28Neurological disorders
    • G01N2800/2814Dementia; Cognitive disorders
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/28Neurological disorders
    • G01N2800/2835Movement disorders, e.g. Parkinson, Huntington, Tourette
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Analytical Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Pathology (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
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  • General Physics & Mathematics (AREA)
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JP2014541200A 2011-11-07 2012-11-07 サンフィリポ症候群のためのバイオマーカーおよびそれらの使用 Pending JP2015504514A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201161556810P 2011-11-07 2011-11-07
US61/556,810 2011-11-07
US201261584165P 2012-01-06 2012-01-06
US61/584,165 2012-01-06
PCT/US2012/063935 WO2013070760A1 (en) 2011-11-07 2012-11-07 Biomarkers for sanfilippo syndrome and uses thereof

Publications (2)

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JP2015504514A true JP2015504514A (ja) 2015-02-12
JP2015504514A5 JP2015504514A5 (enExample) 2015-12-24

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JP2014541200A Pending JP2015504514A (ja) 2011-11-07 2012-11-07 サンフィリポ症候群のためのバイオマーカーおよびそれらの使用

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US (2) US20130121984A1 (enExample)
EP (1) EP2776553B1 (enExample)
JP (1) JP2015504514A (enExample)
CN (1) CN104114686A (enExample)
AU (1) AU2012335890A1 (enExample)
CA (1) CA2854333A1 (enExample)
WO (1) WO2013070760A1 (enExample)

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CA2891522A1 (en) * 2012-12-07 2014-06-12 Shire Human Genetic Therapies, Inc. Methods and compositions for intrathecally administered treatment of mucupolysaccharidosis type iiia
JP2017531632A (ja) * 2014-09-29 2017-10-26 アレクシオン ファーマシューティカルズ, インコーポレイテッド ムコ多糖症iiib型(mpsiiib)を治療する方法
MA40954A (fr) * 2014-11-14 2017-09-19 Shire Human Genetic Therapies Détermination de niveaux de glycosaminoglycane par spectrométrie de masse
WO2017132675A1 (en) * 2016-01-29 2017-08-03 Alexion Pharmaceuticals, Inc. Enzyme replacement therapy in mucopolysaccharidosis iiib patients
EP3684463B1 (en) 2017-09-19 2025-05-14 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement
US11717686B2 (en) 2017-12-04 2023-08-08 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement to facilitate learning and performance
US11318277B2 (en) 2017-12-31 2022-05-03 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement to enhance emotional response
US12280219B2 (en) 2017-12-31 2025-04-22 NeuroLight, Inc. Method and apparatus for neuroenhancement to enhance emotional response
US11364361B2 (en) 2018-04-20 2022-06-21 Neuroenhancement Lab, LLC System and method for inducing sleep by transplanting mental states
EP3849410A4 (en) 2018-09-14 2022-11-02 Neuroenhancement Lab, LLC System and method of improving sleep
US11786694B2 (en) 2019-05-24 2023-10-17 NeuroLight, Inc. Device, method, and app for facilitating sleep
CN112578123B (zh) * 2019-09-27 2022-10-25 成都中医药大学 检测粪钙卫蛋白含量的试剂在制备子宫病变筛查试剂盒中的用途

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005524074A (ja) * 2002-04-30 2005-08-11 生化学工業株式会社 リソソーム病の検出方法
JP2005530157A (ja) * 2002-06-14 2005-10-06 ウイミンズ、アンド、チルドランズ、ホスピトル ムコ多糖症および他の関連障害のオリゴ糖バイオマーカー
JP2007504166A (ja) * 2003-08-29 2007-03-01 バイオマリン ファーマシューティカル インコーポレイテッド 脳およびその他の組織への治療用化合物の送達
US20090092996A1 (en) * 2005-12-08 2009-04-09 Clarke Lorne A Mucopolysaccharidosis (mps) diagnostic methods, systems, kits and assays associated therewith
US20100184013A1 (en) * 2009-01-02 2010-07-22 Zacharon Pharmaceuticals, Inc. Detection of oligosaccharides

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US7015037B1 (en) * 1999-08-05 2006-03-21 Regents Of The University Of Minnesota Multiponent adult stem cells and methods for isolation
US20050055733A1 (en) * 2001-07-06 2005-03-10 Zairen Sun Small intestine and colon genes
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Patent Citations (5)

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Publication number Priority date Publication date Assignee Title
JP2005524074A (ja) * 2002-04-30 2005-08-11 生化学工業株式会社 リソソーム病の検出方法
JP2005530157A (ja) * 2002-06-14 2005-10-06 ウイミンズ、アンド、チルドランズ、ホスピトル ムコ多糖症および他の関連障害のオリゴ糖バイオマーカー
JP2007504166A (ja) * 2003-08-29 2007-03-01 バイオマリン ファーマシューティカル インコーポレイテッド 脳およびその他の組織への治療用化合物の送達
US20090092996A1 (en) * 2005-12-08 2009-04-09 Clarke Lorne A Mucopolysaccharidosis (mps) diagnostic methods, systems, kits and assays associated therewith
US20100184013A1 (en) * 2009-01-02 2010-07-22 Zacharon Pharmaceuticals, Inc. Detection of oligosaccharides

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Title
IGOR NESTRASIL, ET AL.: "Brain Volumes and Cognitive Function in MPS IIIA: Cross-Sectional Study", MOLECULAR GENETICS AND METABOLISM, vol. 102, no. 2, JPN6016039225, February 2011 (2011-02-01), US, pages 32, ISSN: 0003418197 *

Also Published As

Publication number Publication date
EP2776553A1 (en) 2014-09-17
US20170131293A1 (en) 2017-05-11
EP2776553B1 (en) 2018-07-11
CN104114686A (zh) 2014-10-22
EP2776553A4 (en) 2015-05-27
AU2012335890A1 (en) 2014-05-29
CA2854333A1 (en) 2013-05-16
US20130121984A1 (en) 2013-05-16
WO2013070760A1 (en) 2013-05-16

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