HUE046399T2 - Deoxinukleozid terápia kiegyensúlyozatlan nukleotid pool által okozott betegségek, ideértve a mitokondriális DNS depléciós szindrómát, kezelésére - Google Patents

Deoxinukleozid terápia kiegyensúlyozatlan nukleotid pool által okozott betegségek, ideértve a mitokondriális DNS depléciós szindrómát, kezelésére

Info

Publication number
HUE046399T2
HUE046399T2 HUE16812537A HUE16812537A HUE046399T2 HU E046399 T2 HUE046399 T2 HU E046399T2 HU E16812537 A HUE16812537 A HU E16812537A HU E16812537 A HUE16812537 A HU E16812537A HU E046399 T2 HUE046399 T2 HU E046399T2
Authority
HU
Hungary
Prior art keywords
diseases caused
mitochondrial dna
dna depletion
including mitochondrial
nucleotide pools
Prior art date
Application number
HUE16812537A
Other languages
English (en)
Inventor
Michio Hirano
Caterina Garone
Ramon Marti
Original Assignee
Univ Columbia
Fundacio Hospital Univ Vall Dhebron Institut De Recerca
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 Univ Columbia, Fundacio Hospital Univ Vall Dhebron Institut De Recerca filed Critical Univ Columbia
Publication of HUE046399T2 publication Critical patent/HUE046399T2/hu

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/513Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7068Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7068Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
    • A61K31/7072Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid having two oxo groups directly attached to the pyrimidine ring, e.g. uridine, uridylic acid, thymidine, zidovudine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7076Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7076Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
    • A61K31/708Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid having oxo groups directly attached to the purine ring system, e.g. guanosine, guanylic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular 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
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    • 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
    • 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
    • C12Q1/6813Hybridisation assays
    • C12Q1/6827Hybridisation assays for detection of mutation or polymorphism
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

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  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Immunology (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • Zoology (AREA)
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  • Physics & Mathematics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Urology & Nephrology (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Food Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
HUE16812537A 2015-06-17 2016-06-17 Deoxinukleozid terápia kiegyensúlyozatlan nukleotid pool által okozott betegségek, ideértve a mitokondriális DNS depléciós szindrómát, kezelésére HUE046399T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562180914P 2015-06-17 2015-06-17

Publications (1)

Publication Number Publication Date
HUE046399T2 true HUE046399T2 (hu) 2020-03-30

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
HUE16812537A HUE046399T2 (hu) 2015-06-17 2016-06-17 Deoxinukleozid terápia kiegyensúlyozatlan nukleotid pool által okozott betegségek, ideértve a mitokondriális DNS depléciós szindrómát, kezelésére
HUE19156021A HUE050678T2 (hu) 2015-06-17 2016-06-17 Deoxinukleozid terápia ingadozó nukleotid pool által okozott betegségekhez, ideértve a mitokondriális DNS depléciós szindrómát

Family Applications After (1)

Application Number Title Priority Date Filing Date
HUE19156021A HUE050678T2 (hu) 2015-06-17 2016-06-17 Deoxinukleozid terápia ingadozó nukleotid pool által okozott betegségekhez, ideértve a mitokondriális DNS depléciós szindrómát

Country Status (23)

Country Link
US (4) US10471087B2 (hu)
EP (3) EP3310362B1 (hu)
JP (3) JP6599484B2 (hu)
KR (2) KR20220018623A (hu)
CN (2) CN107847512A (hu)
AU (3) AU2016280293B2 (hu)
BR (2) BR112017027079B1 (hu)
CA (1) CA2989653A1 (hu)
CY (2) CY1122605T1 (hu)
DK (2) DK3310362T3 (hu)
ES (2) ES2748556T3 (hu)
HK (1) HK1252133A1 (hu)
HR (2) HRP20191794T1 (hu)
HU (2) HUE046399T2 (hu)
IL (3) IL275256B2 (hu)
LT (2) LT3310362T (hu)
MX (2) MX2020000269A (hu)
PL (2) PL3505174T3 (hu)
PT (2) PT3310362T (hu)
RS (2) RS60572B1 (hu)
RU (2) RU2020112901A (hu)
SI (2) SI3310362T1 (hu)
WO (1) WO2016205671A1 (hu)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10292996B2 (en) * 2015-03-26 2019-05-21 The Trustees Of Columbia University In The City Of New York Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome
WO2016205671A1 (en) * 2015-06-17 2016-12-22 The Trustees Of Columbia University In The City Of New York Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes
KR20180091924A (ko) 2015-12-22 2018-08-16 조게닉스 인터내셔널 리미티드 펜플루라민 조성물 및 그 제조 방법
EP3393470B1 (en) 2015-12-22 2021-01-20 Zogenix International Limited Metabolism resistant fenfluramine analogs and methods of using the same
CN109689103A (zh) 2016-08-24 2019-04-26 周格尼克斯国际有限公司 用于抑制5-ht2b激动剂的形成的制剂及其使用方法
WO2019051097A1 (en) 2017-09-08 2019-03-14 The Regents Of The University Of California RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME
US10682317B2 (en) 2017-09-26 2020-06-16 Zogenix International Limited Ketogenic diet compatible fenfluramine formulation
CA3096264A1 (en) * 2018-04-12 2019-10-17 Modis Therapeutics Inc. Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools
WO2019216919A1 (en) 2018-05-11 2019-11-14 Zogenix International Limited Compositions and methods for treating seizure-induced sudden death
AU2020334060A1 (en) * 2019-08-19 2022-03-24 Zogenix, Inc. Polymorphic forms of deoxycytidine, compositions comprising the same and uses
CA3146835A1 (en) * 2019-09-05 2021-03-11 Mitorainbow Therapeutics, Inc. Treating mitochondrial dna depletion disorders
JP2023518395A (ja) 2020-03-19 2023-05-01 インテリア セラピューティクス,インコーポレイテッド 指向性ゲノム編集のための方法及び組成物
US11612574B2 (en) 2020-07-17 2023-03-28 Zogenix International Limited Method of treating patients infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2)
US20220096514A1 (en) * 2020-09-29 2022-03-31 Zogenix International Limited Methods of treating thymidine kinase 2 deficiency by administering deoxycytidine and deoxythymidine
WO2022145439A1 (ja) * 2020-12-28 2022-07-07 ヤマサ醤油株式会社 筋萎縮抑制剤及び筋萎縮を抑制させる方法
JP2024515025A (ja) * 2021-03-26 2024-04-04 ゾジェニックス,インコーポレイテッド プリン及びピリミジンを含有する水性溶液及びその使用
CA3222484A1 (en) * 2021-06-18 2022-12-22 Daniel DIPIETRO Prodrugs of deoxynucleosides for treatment of mitochondrial diseases caused by unbalanced nucleotide pools
US20240016938A1 (en) * 2022-07-12 2024-01-18 Zogenix Mds, Inc. Methods for increasing the bioavailability of nucleoside medicinal agents

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RU2300381C2 (ru) * 1998-08-10 2007-06-10 Айденикс (Кайман) Лимитед β-L-2'-ДЕЗОКСИНУКЛЕОЗИДЫ ДЛЯ ЛЕЧЕНИЯ ГЕПАТИТА В
US7638501B1 (en) * 1999-02-23 2009-12-29 The Regents Of The University Of California Method of treatment of mitochondrial disorders
ATE275406T1 (de) * 2000-05-26 2004-09-15 Idenix Cayman Ltd Methoden zur behandlung von delta hepatitis virus infektionen mit beta-l-2' deoxy-nucleosiden
WO2012125848A2 (en) * 2011-03-16 2012-09-20 Baylor College Of Medicine A method for comprehensive sequence analysis using deep sequencing technology
US10292996B2 (en) 2015-03-26 2019-05-21 The Trustees Of Columbia University In The City Of New York Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome
RU2745611C2 (ru) * 2015-06-05 2021-03-29 Фундасио Оспиталь Университари Валь Д'Эброн - Институт Де Ресерка Лечение митохондриальных заболеваний
WO2016205671A1 (en) * 2015-06-17 2016-12-22 The Trustees Of Columbia University In The City Of New York Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes

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HK1252133A1 (zh) 2019-05-17
AU2020204042A1 (en) 2020-07-09
AU2021250841A1 (en) 2021-11-04
IL275256B2 (en) 2023-09-01
LT3505174T (lt) 2020-09-10
CY1123107T1 (el) 2021-10-29
EP3310362A4 (en) 2018-12-19
KR20220018623A (ko) 2022-02-15
MX2020000269A (es) 2022-01-03
JP2020019792A (ja) 2020-02-06
RS60572B1 (sr) 2020-08-31
JP6675037B2 (ja) 2020-04-01
US20180133241A1 (en) 2018-05-17
JP2020019791A (ja) 2020-02-06
RU2018101305A (ru) 2019-07-17
US20200038424A1 (en) 2020-02-06
HRP20191794T1 (hr) 2020-02-07
IL256331B (en) 2020-08-31
MX2017016425A (es) 2018-12-11
RU2721492C2 (ru) 2020-05-19
DK3310362T3 (da) 2019-10-07
SI3310362T1 (sl) 2020-02-28
CN116726035A (zh) 2023-09-12
WO2016205671A9 (en) 2018-01-04
BR122020021913B1 (pt) 2021-07-06
CY1122605T1 (el) 2021-03-12
US11110111B2 (en) 2021-09-07
AU2016280293B2 (en) 2020-07-02
IL275255A (en) 2020-07-30
ES2748556T3 (es) 2020-03-17
IL256331A (en) 2018-02-28
HRP20200949T1 (hr) 2020-10-02
JP7036782B2 (ja) 2022-03-15
CN107847512A (zh) 2018-03-27
CA2989653A1 (en) 2016-12-22
PL3310362T3 (pl) 2020-04-30
IL275256B1 (en) 2023-05-01
DK3505174T3 (da) 2020-07-13
AU2020204042B2 (en) 2021-07-15
US10471087B2 (en) 2019-11-12
HUE050678T2 (hu) 2020-12-28
PT3505174T (pt) 2020-07-06
US20210244754A1 (en) 2021-08-12
PT3310362T (pt) 2019-10-29
ES2808148T3 (es) 2021-02-25
EP3569236A1 (en) 2019-11-20
PL3505174T3 (pl) 2020-08-24
BR112017027079B1 (pt) 2021-04-27
SI3505174T1 (sl) 2021-02-26
JP2018521993A (ja) 2018-08-09
IL275256A (en) 2020-07-30
JP6599484B2 (ja) 2019-10-30
US20230277577A1 (en) 2023-09-07
RU2020112901A (ru) 2020-06-05
AU2021250841B2 (en) 2023-02-09
EP3310362A1 (en) 2018-04-25
EP3310362B1 (en) 2019-08-28
EP3505174A1 (en) 2019-07-03
KR20180039624A (ko) 2018-04-18
LT3310362T (lt) 2019-11-11
EP3505174B1 (en) 2020-04-29
RS59724B1 (sr) 2020-01-31
WO2016205671A1 (en) 2016-12-22
AU2016280293A1 (en) 2018-02-08
IL275255B (en) 2021-04-29
US11666592B2 (en) 2023-06-06
RU2018101305A3 (hu) 2019-10-31

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