AR059089A1 - Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal - Google Patents
Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomalInfo
- Publication number
- AR059089A1 AR059089A1 ARP070100239A ARP070100239A AR059089A1 AR 059089 A1 AR059089 A1 AR 059089A1 AR P070100239 A ARP070100239 A AR P070100239A AR P070100239 A ARP070100239 A AR P070100239A AR 059089 A1 AR059089 A1 AR 059089A1
- Authority
- AR
- Argentina
- Prior art keywords
- storage diseases
- enzyme
- lisosomal
- intraventricular administration
- lysosomal storage
- Prior art date
Links
- 102000004190 Enzymes Human genes 0.000 title abstract 2
- 108090000790 Enzymes Proteins 0.000 title abstract 2
- 201000010099 disease Diseases 0.000 title abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title abstract 2
- 238000007914 intraventricular administration Methods 0.000 title abstract 2
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 2
- 230000000694 effects Effects 0.000 abstract 2
- 230000008499 blood brain barrier function Effects 0.000 abstract 1
- 210000001218 blood-brain barrier Anatomy 0.000 abstract 1
- 210000004556 brain Anatomy 0.000 abstract 1
- 230000002950 deficient Effects 0.000 abstract 1
- 210000001835 viscera Anatomy 0.000 abstract 1
Classifications
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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
- 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)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
-
- 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)
-
- 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)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Dermatology (AREA)
- Obesity (AREA)
- Pulmonology (AREA)
- Urology & Nephrology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Infusion, Injection, And Reservoir Apparatuses (AREA)
Abstract
Enfermedades de almacenamiento lisosomal pueden tratarse exitosamente mediante la aplicacion intraventricular de la enzima etiologicamente deficiente en la enfermedad. Para lograr el efecto máximo, la administracion puede realizarse lentamente. Sorprendentemente, se observan efectos en ambos lados de la barrera sangre-cerebro, por lo cual éste es un medio de aplicacion ideal para enfermedades de almacenamiento lisosomal que afectan ambos, el cerebro y organos viscerales.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US76037806P | 2006-01-20 | 2006-01-20 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR059089A1 true AR059089A1 (es) | 2008-03-12 |
Family
ID=38288294
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP070100239A AR059089A1 (es) | 2006-01-20 | 2007-01-19 | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| ARP170102114A AR109170A2 (es) | 2006-01-20 | 2017-07-27 | Esfingomielinasa ácida para uso en la prevención o el tratamiento de la enfermedad de niemann-pick a o b en un paciente |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP170102114A AR109170A2 (es) | 2006-01-20 | 2017-07-27 | Esfingomielinasa ácida para uso en la prevención o el tratamiento de la enfermedad de niemann-pick a o b en un paciente |
Country Status (19)
| Country | Link |
|---|---|
| US (4) | US8926967B2 (es) |
| EP (3) | EP3517124A1 (es) |
| JP (1) | JP5681345B2 (es) |
| CN (1) | CN101431960A (es) |
| AR (2) | AR059089A1 (es) |
| BR (1) | BRPI0706699A2 (es) |
| CA (2) | CA2966155A1 (es) |
| DK (2) | DK2666476T3 (es) |
| ES (2) | ES2526710T3 (es) |
| HU (1) | HUE041840T2 (es) |
| IL (2) | IL192740A (es) |
| LT (1) | LT2666476T (es) |
| MX (1) | MX349749B (es) |
| PL (2) | PL1984018T3 (es) |
| PT (2) | PT1984018E (es) |
| RU (3) | RU2529830C2 (es) |
| SI (2) | SI1984018T1 (es) |
| TR (1) | TR201903855T4 (es) |
| WO (1) | WO2007084737A2 (es) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| HUE040490T2 (hu) * | 2006-02-09 | 2019-03-28 | Genzyme Corp | Lassú intraventrikuláris szállítás |
| EP2154969B1 (en) | 2007-05-16 | 2015-11-18 | The Brigham and Women's Hospital, Inc. | Treatment of synucleinopathies |
| CN102369023A (zh) * | 2008-12-16 | 2012-03-07 | 建新公司 | 寡糖-蛋白缀合物 |
| HRP20250897T1 (hr) | 2009-05-02 | 2025-09-26 | Genzyme Corporation | Genska terapija za neurodegenerativne poremećaje |
| SI3998078T1 (sl) * | 2009-08-28 | 2026-02-27 | Genzyme Corporation | Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze |
| FI3205351T3 (fi) | 2010-04-23 | 2023-07-06 | Alexion Pharma Inc | Lysosomaalisen kertymätaudin entsyymi |
| US20110318324A1 (en) * | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of b-galactocerebrosidase |
| KR20230159646A (ko) | 2010-06-25 | 2023-11-21 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| JP6073783B2 (ja) | 2010-06-25 | 2017-02-01 | 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 | ヘパランn−スルファターゼのcns送達のための方法および組成物 |
| PT3103469T (pt) | 2010-06-25 | 2021-03-04 | Shire Human Genetic Therapies | Administração de agentes terapêuticos no snc |
| LT2588130T (lt) * | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas cns |
| HRP20191923T1 (hr) * | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku arilsulfataze a u cns |
| JP2012062312A (ja) * | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | ハンター症候群の治療剤 |
| KR20150038636A (ko) | 2010-09-09 | 2015-04-08 | 시나게바 바이오파르마, 코포레이션 | 환자에서 리소좀 산 리파제 결핍증의 치료를 위한 리소좀 산 리파제의 용도 |
| US9387236B2 (en) * | 2011-06-10 | 2016-07-12 | Prothera Inc. | Pharmaceutical compositions containing protease and methods for the treatment of lysosomal storage diseases |
| US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| US9682129B2 (en) | 2011-12-23 | 2017-06-20 | Shire Human Genetic Therapies, Inc. | Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase |
| EA039246B1 (ru) * | 2012-12-06 | 2021-12-22 | Шир Хьюман Дженетик Терапис, Инк. | Способ лечения когнитивных нарушений, связанных с синдромом хантера |
| AU2014218854B2 (en) | 2013-02-20 | 2019-01-24 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Pompe disease |
| ES2762608T3 (es) | 2013-06-07 | 2020-05-25 | Genzyme Corp | Marcador para trastornos de esfingomielinasa ácida y usos del mismo |
| EP3088518A4 (en) | 2013-12-25 | 2017-09-20 | JCR Pharmaceuticals Co., Ltd. | Novel anti-transferrin receptor antibody that passes through blood-brain barrier |
| US10357542B2 (en) | 2014-02-04 | 2019-07-23 | New York University | Progranulin (PGRN) and its derivatives for diagnosis and treatment of lysosomal storage diseases |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| AU2016283344B2 (en) | 2015-06-24 | 2022-08-04 | Jcr Pharmaceuticals Co., Ltd. | Fusion protein containing BDNF |
| EA039366B1 (ru) | 2015-06-24 | 2022-01-19 | Джей Си Ар ФАРМАСЬЮТИКАЛЗ КО., ЛТД. | Антитело к рецептору трансферрина человека, проникающее через гематоэнцефалический барьер |
| WO2017024137A1 (en) * | 2015-08-04 | 2017-02-09 | New York University | Progranulin (pgrn) fragments and derivatives for treatment or alleviation of lysosomal storage diseases |
| RU2634113C1 (ru) * | 2016-11-18 | 2017-10-24 | Государственное бюджетное учреждение "Академия наук Республики Саха (Якутия)" (ГБУ АН РС(Я)) | Способ фиксации головного мозга ископаемого позднеплейстоценового млекопитающего |
| KR102497564B1 (ko) | 2016-12-26 | 2023-02-07 | 제이씨알 파마 가부시키가이샤 | 혈액뇌관문을 통과하는 신규한 항인간 트랜스페린 수용체 항체 |
| CA3052936A1 (en) | 2016-12-26 | 2018-07-05 | Jcr Pharmaceuticals Co., Ltd. | Fusion protein of bdnf and anti-transferrin receptor antibody |
| CR20200129A (es) | 2017-10-02 | 2020-08-22 | Denali Therapeutics Inc | Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas |
| PY1939780A (es) * | 2018-05-25 | 2019-12-04 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
| US20220125892A1 (en) | 2019-02-01 | 2022-04-28 | OXYRANE UK Ltd. | Glucocerebrosidase polypeptides |
| RU2723187C1 (ru) * | 2019-10-04 | 2020-06-09 | федеральное государственное автономное образовательное учреждение высшего образования "Казанский (Приволжский) федеральный университет" (ФГАОУ ВО КФУ) | Генетическая кассета, содержащая кодон-оптимизированные нуклеотидные последовательности генов HEXA и HEXВ, и фармацевтическая композиция для лечения болезни Тея-Сакса |
| EP4284442A4 (en) * | 2021-02-01 | 2025-04-23 | The Trustees of The University of Pennsylvania | COMPOSITIONS AND METHODS FOR TREATING NIEMANN PICK DISEASE TYPE A |
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| CA2189067A1 (en) | 1994-04-28 | 1995-11-09 | Gary J. Nabel | Gene delivery vector using plasmid dna packaged into an adenovirus and a packaging cell line |
| AU5551996A (en) | 1995-04-17 | 1996-11-07 | Board Of Regents, The University Of Texas System | An adenovirus helper-virus system |
| IL160406A0 (en) | 1995-06-15 | 2004-07-25 | Crucell Holland Bv | A cell harbouring nucleic acid encoding adenoritus e1a and e1b gene products |
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| AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| AR059088A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una proteina para esclerosis lateral amiotrofica |
| HUE040490T2 (hu) | 2006-02-09 | 2019-03-28 | Genzyme Corp | Lassú intraventrikuláris szállítás |
| ES2635726T3 (es) * | 2007-06-06 | 2017-10-04 | Genzyme Corporation | Terapia génica para enfermedades de almacenamiento lisosómico |
-
2007
- 2007-01-19 AR ARP070100239A patent/AR059089A1/es not_active Application Discontinuation
- 2007-01-22 TR TR2019/03855T patent/TR201903855T4/tr unknown
- 2007-01-22 PT PT77168581T patent/PT1984018E/pt unknown
- 2007-01-22 SI SI200731529T patent/SI1984018T1/sl unknown
- 2007-01-22 RU RU2008134120/15A patent/RU2529830C2/ru not_active IP Right Cessation
- 2007-01-22 EP EP18212320.8A patent/EP3517124A1/en not_active Withdrawn
- 2007-01-22 JP JP2008551442A patent/JP5681345B2/ja not_active Expired - Fee Related
- 2007-01-22 PT PT13173895T patent/PT2666476T/pt unknown
- 2007-01-22 ES ES07716858.1T patent/ES2526710T3/es active Active
- 2007-01-22 CA CA2966155A patent/CA2966155A1/en not_active Abandoned
- 2007-01-22 LT LTEP13173895.7T patent/LT2666476T/lt unknown
- 2007-01-22 MX MX2013012372A patent/MX349749B/es unknown
- 2007-01-22 PL PL07716858T patent/PL1984018T3/pl unknown
- 2007-01-22 DK DK13173895.7T patent/DK2666476T3/en active
- 2007-01-22 CA CA2636991A patent/CA2636991C/en not_active Expired - Fee Related
- 2007-01-22 EP EP07716858.1A patent/EP1984018B1/en not_active Not-in-force
- 2007-01-22 EP EP13173895.7A patent/EP2666476B1/en active Active
- 2007-01-22 CN CNA2007800069255A patent/CN101431960A/zh active Pending
- 2007-01-22 BR BRPI0706699-6A patent/BRPI0706699A2/pt not_active Application Discontinuation
- 2007-01-22 ES ES13173895T patent/ES2716206T3/es active Active
- 2007-01-22 DK DK07716858.1T patent/DK1984018T3/da active
- 2007-01-22 SI SI200732098T patent/SI2666476T1/sl unknown
- 2007-01-22 PL PL13173895T patent/PL2666476T3/pl unknown
- 2007-01-22 HU HUE13173895A patent/HUE041840T2/hu unknown
- 2007-01-22 WO PCT/US2007/001566 patent/WO2007084737A2/en not_active Ceased
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2008
- 2008-07-10 IL IL192740A patent/IL192740A/en not_active IP Right Cessation
- 2008-07-18 US US12/175,610 patent/US8926967B2/en active Active
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2014
- 2014-07-16 RU RU2014129319A patent/RU2665381C2/ru not_active IP Right Cessation
- 2014-12-03 US US14/559,851 patent/US10080783B2/en active Active
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2017
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- 2017-07-27 AR ARP170102114A patent/AR109170A2/es unknown
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2018
- 2018-08-15 RU RU2018129747A patent/RU2018129747A/ru not_active Application Discontinuation
- 2018-08-23 US US16/111,009 patent/US20190083582A1/en not_active Abandoned
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2020
- 2020-12-29 US US17/137,060 patent/US20210228692A1/en not_active Abandoned
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