ES2523024T3 - Tratamiento de glucogenosis de tipo II - Google Patents
Tratamiento de glucogenosis de tipo II Download PDFInfo
- Publication number
- ES2523024T3 ES2523024T3 ES07001091.3T ES07001091T ES2523024T3 ES 2523024 T3 ES2523024 T3 ES 2523024T3 ES 07001091 T ES07001091 T ES 07001091T ES 2523024 T3 ES2523024 T3 ES 2523024T3
- Authority
- ES
- Spain
- Prior art keywords
- treatment
- type
- glycogenosis
- months
- storage disease
- Prior art date
- Legal status (The legal status 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 status listed.)
- Expired - Lifetime
Links
Classifications
-
- 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
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
-
- 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
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- 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
- A61P9/02—Non-specific cardiovascular stimulants, e.g. drugs for syncope, antihypotensives
-
- 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
- A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
-
- 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)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Immunology (AREA)
- Cardiology (AREA)
- Diabetes (AREA)
- Heart & Thoracic Surgery (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Genetics & Genomics (AREA)
- General Engineering & Computer Science (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Neurology (AREA)
- Hospice & Palliative Care (AREA)
- Emergency Medicine (AREA)
- Endocrinology (AREA)
- Transplantation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
α-Glucosidasa ácida (GAA) humana recombinante producida en un cultivo celular de ovario de hámster chino para uso en un método de tratamiento de la glucogenosis de tipo II en un ser humano, o para uso en un método de tratamiento de la cardiomiopatía asociada con la glucogenosis de tipo II en un ser humano, donde el ser humano es CRIM (material immunoreactivo de reacción cruzada)-positivo para la α-glucosidasa ácida endógena.
Description
E07001091
03-11-2014
Tabla 2. Datos clínicos de referencia en 3 pacientes con enfermedad de Pompe infantil
- Número depaciente/sexo
- Origen étnico Edad inicioGAArh Estado cardiaco Función pulmonar Desarrollomotor(puntuaciónAIMS) Actividad GAAen fibroblastosde la piel (% denormal) EstadoCRIM* Edadactual
- Paciente1/masculino
- Caucásico 4 meses Cardiomiopatíasevera; estadopost ataquecardiaco Límite normal,compresiónbronquio principalizquierdo por unamarcadacardiomegalia,desaturación O2 <<5º% 0,84% Negativo 29 meses
- Paciente2/masculino
- Afro-Americano 3 meses Cardiomiopatíamoderada Desaturación O2durante el llanto <5º% 0,57% Negativo 25 meses
- Paciente3/masculino
- Caucásico 2½ meses Cardiomiopatíalímite normal <<5º% 0,69% Positivo 23 meses
*CRIM = material inmunoreactivo de reacción cruzada
7
Claims (1)
-
imagen1
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US21923700P | 2000-07-18 | 2000-07-18 | |
US219237P | 2000-07-18 |
Publications (1)
Publication Number | Publication Date |
---|---|
ES2523024T3 true ES2523024T3 (es) | 2014-11-20 |
Family
ID=22818451
Family Applications (5)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ES18194771T Expired - Lifetime ES2799882T3 (es) | 2000-07-18 | 2001-07-10 | Tratamiento de glucogenosis de tipo II |
ES01951000T Expired - Lifetime ES2277931T3 (es) | 2000-07-18 | 2001-07-10 | Tratamiento de la glucogenesis de tipo ii. |
ES16178390T Expired - Lifetime ES2700865T3 (es) | 2000-07-18 | 2001-07-10 | Tratamiento de glucogenosis de tipo II |
ES14167882.1T Expired - Lifetime ES2599401T3 (es) | 2000-07-18 | 2001-07-10 | Tratamiento de glucogenosis de tipo II |
ES07001091.3T Expired - Lifetime ES2523024T3 (es) | 2000-07-18 | 2001-07-10 | Tratamiento de glucogenosis de tipo II |
Family Applications Before (4)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ES18194771T Expired - Lifetime ES2799882T3 (es) | 2000-07-18 | 2001-07-10 | Tratamiento de glucogenosis de tipo II |
ES01951000T Expired - Lifetime ES2277931T3 (es) | 2000-07-18 | 2001-07-10 | Tratamiento de la glucogenesis de tipo ii. |
ES16178390T Expired - Lifetime ES2700865T3 (es) | 2000-07-18 | 2001-07-10 | Tratamiento de glucogenosis de tipo II |
ES14167882.1T Expired - Lifetime ES2599401T3 (es) | 2000-07-18 | 2001-07-10 | Tratamiento de glucogenosis de tipo II |
Country Status (18)
Country | Link |
---|---|
US (10) | US7056712B2 (es) |
EP (4) | EP3449934B1 (es) |
JP (10) | JP5113312B2 (es) |
AT (1) | ATE355075T1 (es) |
AU (3) | AU2001271941B2 (es) |
BR (1) | BR0113006A (es) |
CA (1) | CA2416492C (es) |
CY (2) | CY1120977T1 (es) |
DE (1) | DE60126947T2 (es) |
DK (5) | DK1782825T3 (es) |
ES (5) | ES2799882T3 (es) |
HK (3) | HK1054690A1 (es) |
LT (3) | LT3108895T (es) |
MX (1) | MXPA03000474A (es) |
PT (5) | PT3108895T (es) |
SI (3) | SI3449934T1 (es) |
TW (1) | TWI280882B (es) |
WO (1) | WO2002005841A1 (es) |
Families Citing this family (30)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
ES2799882T3 (es) * | 2000-07-18 | 2020-12-22 | Univ Duke | Tratamiento de glucogenosis de tipo II |
US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
CA2445577C (en) * | 2001-04-30 | 2012-07-03 | Symbiontics, Inc. | Subcellular targeting of therapeutic proteins |
US20040005309A1 (en) * | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
US7629309B2 (en) * | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
US20030072761A1 (en) * | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
ATE521701T1 (de) | 2003-01-22 | 2011-09-15 | Univ Duke | Verbesserte konstrukte zur expression lysosomaler polypeptide |
US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
US7129049B2 (en) * | 2003-12-22 | 2006-10-31 | Regents Of The University Of Minnesota | Method of detecting equine glycogen storage disease IV |
WO2005078077A2 (en) * | 2004-02-10 | 2005-08-25 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
EP1773401B1 (en) * | 2004-06-21 | 2013-01-02 | Medtronic, Inc. | Medical systems and methods for delivering compositions to cells |
JP2010509344A (ja) * | 2006-11-13 | 2010-03-25 | ザイストール セラピューティクス, インコーポレイテッド | ポンペ病を治療するための方法 |
WO2009075815A1 (en) | 2007-12-07 | 2009-06-18 | Duke University | Immunomodulating gene therapy |
DK2279210T3 (en) | 2008-05-07 | 2017-07-24 | Biomarin Pharm Inc | Lysosomal targeting peptides and uses thereof |
WO2010005565A2 (en) | 2008-07-08 | 2010-01-14 | Duke University | Method of treating glycogen storage disease |
ES2569514T3 (es) | 2009-06-17 | 2016-05-11 | Biomarin Pharmaceutical Inc. | Formulaciones para enzimas lisosómicas |
US8809282B2 (en) | 2010-05-06 | 2014-08-19 | Duke University | Method of reducing titers of antibodies specific for a therapeutic agent |
AU2014218854B2 (en) | 2013-02-20 | 2019-01-24 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Pompe disease |
CN115120745A (zh) * | 2013-05-15 | 2022-09-30 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经***转移 |
FI3201320T3 (fi) | 2014-09-30 | 2024-01-08 | Amicus Therapeutics Inc | Erittäin tehokas hapan alfa-glukosidaasi parannetuilla hiilihydraateilla |
JP6821192B2 (ja) * | 2015-05-07 | 2021-01-27 | 国立大学法人 東京大学 | ポリイオンコンプレックス型ポリマーソームを用いたナノリアクタとその製造方法 |
MA44747A (fr) * | 2015-08-31 | 2019-03-06 | Univ Duke | Méthodes et compositions pour le traitement de troubles associés à une accumulation du glycogène cytoplasmique |
US10940125B2 (en) | 2015-09-18 | 2021-03-09 | Duke University | Methods and compositions for the treatment of steatosis-associated disorders |
KR102510941B1 (ko) | 2015-12-30 | 2023-03-20 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
KR20220145918A (ko) | 2016-03-30 | 2022-10-31 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 고 m6p 재조합 단백질의 선택 방법 |
CA3019128A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
IL305449A (en) | 2016-04-15 | 2023-10-01 | Univ Pennsylvania | Gene therapy for the treatment of type II mucositis |
CA3076036A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
WO2020163480A1 (en) * | 2019-02-05 | 2020-08-13 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase and uses thereof |
Family Cites Families (11)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US3063911A (en) * | 1961-04-03 | 1962-11-13 | Taisho Pharmaceutical Co Ltd | Acid-stable digestive enzyme preparation and process of making same |
US5464778A (en) | 1989-03-08 | 1995-11-07 | Board Of Regents Of The University Of Oklahoma | Glycoprotein ligand for P-selectin and methods of use thereof |
US5356804A (en) * | 1990-10-24 | 1994-10-18 | Mount Sinai School Of Medicine Of The City Of New York | Cloning and expression of biologically active human α-galactosidase A |
US6118045A (en) | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
IL139616A0 (en) | 1998-05-13 | 2002-02-10 | Harbor Ucla | Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating, diseases caused by deficiencies thereof |
KR20010101131A (ko) * | 1998-12-07 | 2001-11-14 | 추후기재 | 폼페병의 치료 방법 |
US6534300B1 (en) | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
ES2799882T3 (es) * | 2000-07-18 | 2020-12-22 | Univ Duke | Tratamiento de glucogenosis de tipo II |
US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
WO2005078077A2 (en) * | 2004-02-10 | 2005-08-25 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
US8470552B2 (en) | 2009-10-12 | 2013-06-25 | Keck Graduate Institute | Strategy to reduce lactic acid production and control PH in animal cell culture |
-
2001
- 2001-07-10 ES ES18194771T patent/ES2799882T3/es not_active Expired - Lifetime
- 2001-07-10 WO PCT/US2001/021651 patent/WO2002005841A1/en active IP Right Grant
- 2001-07-10 DK DK07001091.3T patent/DK1782825T3/da active
- 2001-07-10 PT PT16178390T patent/PT3108895T/pt unknown
- 2001-07-10 ES ES01951000T patent/ES2277931T3/es not_active Expired - Lifetime
- 2001-07-10 LT LTEP16178390.7T patent/LT3108895T/lt unknown
- 2001-07-10 ES ES16178390T patent/ES2700865T3/es not_active Expired - Lifetime
- 2001-07-10 AT AT01951000T patent/ATE355075T1/de active
- 2001-07-10 SI SI200131073T patent/SI3449934T1/sl unknown
- 2001-07-10 DK DK14167882.1T patent/DK2767291T3/en active
- 2001-07-10 ES ES14167882.1T patent/ES2599401T3/es not_active Expired - Lifetime
- 2001-07-10 DK DK01951000T patent/DK1301201T3/da active
- 2001-07-10 MX MXPA03000474A patent/MXPA03000474A/es active IP Right Grant
- 2001-07-10 US US09/902,461 patent/US7056712B2/en not_active Expired - Lifetime
- 2001-07-10 PT PT70010913T patent/PT1782825E/pt unknown
- 2001-07-10 EP EP18194771.4A patent/EP3449934B1/en not_active Expired - Lifetime
- 2001-07-10 CA CA002416492A patent/CA2416492C/en not_active Expired - Lifetime
- 2001-07-10 JP JP2002511773A patent/JP5113312B2/ja not_active Expired - Lifetime
- 2001-07-10 DK DK16178390.7T patent/DK3108895T3/en active
- 2001-07-10 AU AU2001271941A patent/AU2001271941B2/en not_active Expired
- 2001-07-10 LT LTEP18194771.4T patent/LT3449934T/lt unknown
- 2001-07-10 LT LTEP14167882.1T patent/LT2767291T/lt unknown
- 2001-07-10 PT PT181947714T patent/PT3449934T/pt unknown
- 2001-07-10 PT PT01951000T patent/PT1301201E/pt unknown
- 2001-07-10 DK DK18194771.4T patent/DK3449934T3/da active
- 2001-07-10 SI SI200131057A patent/SI2767291T1/sl unknown
- 2001-07-10 AU AU7194101A patent/AU7194101A/xx active Pending
- 2001-07-10 EP EP14167882.1A patent/EP2767291B1/en not_active Expired - Lifetime
- 2001-07-10 DE DE60126947T patent/DE60126947T2/de not_active Expired - Lifetime
- 2001-07-10 TW TW090116828A patent/TWI280882B/zh not_active IP Right Cessation
- 2001-07-10 BR BR0113006-4A patent/BR0113006A/pt not_active Application Discontinuation
- 2001-07-10 ES ES07001091.3T patent/ES2523024T3/es not_active Expired - Lifetime
- 2001-07-10 SI SI200131068T patent/SI3108895T1/sl unknown
- 2001-07-10 EP EP01951000A patent/EP1301201B1/en not_active Revoked
- 2001-07-10 PT PT141678821T patent/PT2767291T/pt unknown
- 2001-07-10 EP EP16178390.7A patent/EP3108895B1/en not_active Expired - Lifetime
-
2003
- 2003-09-27 HK HK03107002A patent/HK1054690A1/xx not_active IP Right Cessation
-
2005
- 2005-01-20 US US11/039,281 patent/US20050123531A1/en not_active Abandoned
-
2007
- 2007-08-13 US US11/889,457 patent/US20080175833A1/en not_active Abandoned
- 2007-11-03 HK HK14112435.2A patent/HK1197656A1/zh not_active IP Right Cessation
- 2007-11-03 HK HK07111945.6A patent/HK1103363A1/xx not_active IP Right Cessation
- 2007-12-03 JP JP2007312804A patent/JP2008081507A/ja active Pending
-
2009
- 2009-10-22 US US12/604,267 patent/US20100254966A1/en not_active Abandoned
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2010
- 2010-02-10 AU AU2010200487A patent/AU2010200487A1/en not_active Ceased
-
2011
- 2011-03-30 US US13/064,556 patent/US20120058132A1/en not_active Abandoned
- 2011-08-09 JP JP2011173569A patent/JP2012006953A/ja active Pending
-
2012
- 2012-08-23 US US13/593,148 patent/US8900552B2/en not_active Expired - Fee Related
-
2013
- 2013-08-05 JP JP2013162201A patent/JP5792774B2/ja not_active Expired - Lifetime
-
2014
- 2014-07-09 JP JP2014141336A patent/JP2014185187A/ja active Pending
- 2014-09-12 US US14/485,071 patent/US9370556B2/en not_active Expired - Lifetime
-
2016
- 2016-01-14 JP JP2016005132A patent/JP6163216B2/ja not_active Expired - Lifetime
- 2016-03-10 US US15/066,211 patent/US9907839B2/en not_active Expired - Fee Related
-
2017
- 2017-05-19 JP JP2017099515A patent/JP2017137357A/ja active Pending
- 2017-10-17 JP JP2017201250A patent/JP2018002749A/ja active Pending
-
2018
- 2018-01-19 US US15/876,086 patent/US10792341B2/en not_active Expired - Lifetime
- 2018-09-13 JP JP2018171154A patent/JP2018199721A/ja active Pending
- 2018-12-07 CY CY181101308T patent/CY1120977T1/el unknown
-
2019
- 2019-11-08 JP JP2019202851A patent/JP2020019822A/ja active Pending
-
2020
- 2020-06-24 CY CY20201100587T patent/CY1123084T1/el unknown
- 2020-07-27 US US16/940,054 patent/US20210169996A1/en not_active Abandoned
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