ES2523024T3 - Tratamiento de glucogenosis de tipo II - Google Patents

Tratamiento de glucogenosis de tipo II Download PDF

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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
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treatment
type
glycogenosis
months
storage disease
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ES07001091.3T
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English (en)
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Yuan-Tsong Chen
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Duke University
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Duke University
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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)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • 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
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • 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
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/02Non-specific cardiovascular stimulants, e.g. drugs for syncope, antihypotensives
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102Alpha-glucosidase (3.2.1.20)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides

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  • 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

imagen1
imagen2
imagen3
imagen4
imagen5
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
imagen6
imagen7
imagen8
imagen9

Claims (1)

  1. imagen1
ES07001091.3T 2000-07-18 2001-07-10 Tratamiento de glucogenosis de tipo II Expired - Lifetime ES2523024T3 (es)

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)

* Cited by examiner, † Cited by third party
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

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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
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SI3108895T1 (sl) 2019-01-31
JP2020019822A (ja) 2020-02-06
EP2767291B1 (en) 2016-09-14
BR0113006A (pt) 2004-10-19
US9907839B2 (en) 2018-03-06
JP5792774B2 (ja) 2015-10-14
HK1103363A1 (en) 2007-12-21
DK1782825T3 (da) 2014-11-03
MXPA03000474A (es) 2003-10-06
US20020110551A1 (en) 2002-08-15
ATE355075T1 (de) 2006-03-15
DK1301201T3 (da) 2007-04-02
LT2767291T (lt) 2016-11-25
EP1301201A1 (en) 2003-04-16
PT3449934T (pt) 2020-06-25
US20150004152A1 (en) 2015-01-01
US20050123531A1 (en) 2005-06-09
US8900552B2 (en) 2014-12-02
JP2014185187A (ja) 2014-10-02
JP2016056206A (ja) 2016-04-21
EP3108895B1 (en) 2018-09-19
DK3449934T3 (da) 2020-06-29
PT1782825E (pt) 2014-11-04
CY1123084T1 (el) 2021-10-29
WO2002005841A1 (en) 2002-01-24
JP2012006953A (ja) 2012-01-12
ES2700865T3 (es) 2019-02-19
US20210169996A1 (en) 2021-06-10
US20130195834A1 (en) 2013-08-01
JP2008081507A (ja) 2008-04-10
EP3449934A1 (en) 2019-03-06
DK2767291T3 (en) 2016-12-05
JP2013231081A (ja) 2013-11-14
ES2277931T3 (es) 2007-08-01
JP6163216B2 (ja) 2017-07-12
US20080175833A1 (en) 2008-07-24
EP2767291A2 (en) 2014-08-20
EP3108895A1 (en) 2016-12-28
PT3108895T (pt) 2018-12-18
US20120058132A1 (en) 2012-03-08
DE60126947D1 (de) 2007-04-12
US20180185457A1 (en) 2018-07-05
US9370556B2 (en) 2016-06-21
ES2799882T3 (es) 2020-12-22
EP1301201B1 (en) 2007-02-28
DK3108895T3 (en) 2018-11-26
CA2416492C (en) 2008-04-29
ES2599401T3 (es) 2017-02-01
US7056712B2 (en) 2006-06-06
US20100254966A1 (en) 2010-10-07
SI2767291T1 (sl) 2016-11-30
AU2001271941B2 (en) 2007-03-01
SI3449934T1 (sl) 2020-09-30
AU2010200487A1 (en) 2010-03-04
US20160184410A1 (en) 2016-06-30
JP5113312B2 (ja) 2013-01-09
AU7194101A (en) 2002-01-30
EP3449934B1 (en) 2020-05-20
DE60126947T2 (de) 2007-10-31
CY1120977T1 (el) 2019-12-11
HK1054690A1 (en) 2003-12-12
CA2416492A1 (en) 2002-01-24
JP2004503598A (ja) 2004-02-05
PT2767291T (pt) 2016-10-25
JP2018002749A (ja) 2018-01-11
LT3108895T (lt) 2018-11-26
HK1197656A1 (zh) 2015-02-06
JP2017137357A (ja) 2017-08-10
LT3449934T (lt) 2020-08-10
EP2767291A3 (en) 2014-10-08
US10792341B2 (en) 2020-10-06
PT1301201E (pt) 2007-03-30
JP2018199721A (ja) 2018-12-20
TWI280882B (en) 2007-05-11

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