PT3470077T - Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas - Google Patents
Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonasInfo
- Publication number
- PT3470077T PT3470077T PT182019604T PT18201960T PT3470077T PT 3470077 T PT3470077 T PT 3470077T PT 182019604 T PT182019604 T PT 182019604T PT 18201960 T PT18201960 T PT 18201960T PT 3470077 T PT3470077 T PT 3470077T
- Authority
- PT
- Portugal
- Prior art keywords
- diseases
- pharmacological chaperone
- predict response
- chaperone treatment
- treatment
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
-
- 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
- A61P3/06—Antihyperlipidemics
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING 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
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical 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
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/435—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
- G01N2333/46—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans from vertebrates
- G01N2333/47—Assays involving proteins of known structure or function as defined in the subgroups
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/90—Enzymes; Proenzymes
- G01N2333/914—Hydrolases (3)
- G01N2333/924—Hydrolases (3) acting on glycosyl compounds (3.2)
- G01N2333/94—Hydrolases (3) acting on glycosyl compounds (3.2) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/38—Pediatrics
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US2814108P | 2008-02-12 | 2008-02-12 | |
US3568408P | 2008-03-11 | 2008-03-11 | |
US9363108P | 2008-09-02 | 2008-09-02 | |
US11349608P | 2008-11-11 | 2008-11-11 |
Publications (1)
Publication Number | Publication Date |
---|---|
PT3470077T true PT3470077T (pt) | 2020-11-30 |
Family
ID=40957500
Family Applications (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PT97101786T PT2252313E (pt) | 2008-02-12 | 2009-02-12 | Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas |
PT15162630T PT2946785T (pt) | 2008-02-12 | 2009-02-12 | Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas |
PT182019604T PT3470077T (pt) | 2008-02-12 | 2009-02-12 | Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas |
Family Applications Before (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PT97101786T PT2252313E (pt) | 2008-02-12 | 2009-02-12 | Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas |
PT15162630T PT2946785T (pt) | 2008-02-12 | 2009-02-12 | Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas |
Country Status (16)
Country | Link |
---|---|
US (7) | US8592362B2 (pt) |
EP (4) | EP2252313B1 (pt) |
JP (6) | JP5844045B2 (pt) |
AU (6) | AU2009214648B2 (pt) |
CA (1) | CA2715407C (pt) |
CY (3) | CY1116466T1 (pt) |
DK (3) | DK2252313T3 (pt) |
ES (3) | ES2716502T3 (pt) |
HR (3) | HRP20150728T1 (pt) |
HU (3) | HUE042882T2 (pt) |
LT (2) | LT3470077T (pt) |
MX (1) | MX2010008835A (pt) |
PL (3) | PL2252313T3 (pt) |
PT (3) | PT2252313E (pt) |
SI (3) | SI2252313T1 (pt) |
WO (1) | WO2009102895A2 (pt) |
Families Citing this family (37)
Publication number | Priority date | Publication date | Assignee | Title |
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ES2709445T3 (es) | 2006-05-16 | 2019-04-16 | Amicus Therapeutics Inc | Opciones de tratamiento para la enfermedad de Fabry |
DK2252313T3 (en) | 2008-02-12 | 2015-07-13 | Amicus Therapeutics Inc | METHOD TO PREDICT response to Pharmacological chaperone TREATMENT OF DISEASES |
US20110136151A1 (en) * | 2008-03-12 | 2011-06-09 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for pompe disease |
TW201117810A (en) * | 2009-10-01 | 2011-06-01 | Baylor Res Inst | Treatment of male-pattern baldness by local induction of the metabolic defect of fabry disease |
US20120283290A1 (en) * | 2011-05-06 | 2012-11-08 | Amicus Therapeutics Inc. | Quantitation of gl3 in urine |
PL3201320T3 (pl) | 2014-09-30 | 2024-03-18 | Amicus Therapeutics, Inc. | Bardzo silnie działająca kwasowa alfa-glukozydaza ze zwiększoną zawartością węglowodanów |
AU2016233135B2 (en) | 2015-03-19 | 2021-07-08 | Translate Bio, Inc. | mRNA therapy for pompe disease |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
BR112018013151A2 (pt) | 2015-12-30 | 2018-12-18 | Amicus Therapeutics Inc | alfa-glicosidase ácida aumentada para o tratamento da doença de pompe |
SI3957320T1 (sl) | 2015-12-30 | 2024-01-31 | Amicus Therapeutics, Inc., | Dopolnjena kisla alfa glukozidaza za zdravljenje Pompejeve bolezni |
EP3432882B1 (en) * | 2016-03-22 | 2021-09-01 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having the g9331a mutation in the gla gene |
WO2017173059A1 (en) * | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Method for selection of high m6p recombinant proteins |
WO2017173060A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
TWI813388B (zh) | 2016-03-30 | 2023-08-21 | 美商阿米庫斯醫療股份有限公司 | 包含重組酸性α-葡萄糖苷酶之調配物 |
US20190183869A1 (en) | 2016-07-19 | 2019-06-20 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naïve and ert-experienced patients |
JP6546135B2 (ja) | 2016-08-24 | 2019-07-17 | 株式会社ノリタケカンパニーリミテド | 積層造形焼成体、積層造形焼成体の製造方法および積層造形焼成体製造用キット |
EP3528852A4 (en) * | 2016-10-20 | 2020-06-03 | Sangamo Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR THE TREATMENT OF FABRY'S DISEASE |
EA039750B1 (ru) * | 2017-03-30 | 2022-03-10 | Амикус Терапьютикс, Инк. | Способ отбора рекомбинантных белков с высоким содержанием m6p |
AU2018270925A1 (en) | 2017-05-15 | 2019-11-28 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase |
CA3065298A1 (en) | 2017-05-30 | 2018-12-06 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
CN111278438A (zh) | 2017-05-30 | 2020-06-12 | 阿米库斯治疗学公司 | 治疗具有肾损害的法布里患者的方法 |
SG11202000423XA (en) | 2017-07-19 | 2020-02-27 | Amicus Therapeutics Inc | Treatment of fabry disease in ert-naïve and ert-experienced patients |
HRP20221366T1 (hr) | 2017-08-28 | 2023-01-06 | Amicus Therapeutics, Inc. | Postupci poboljšanja i/ili stabilizacije srčane funkcije kod bolesnika s fabrijevom bolešću |
WO2019157047A1 (en) | 2018-02-06 | 2019-08-15 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
DK3749308T3 (da) | 2018-02-06 | 2024-01-22 | Amicus Therapeutics Inc | Behandling af patienter med klassisk fabrys sygdom med migalastat |
WO2020023390A1 (en) | 2018-07-25 | 2020-01-30 | Modernatx, Inc. | Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders |
BR112021003137A2 (pt) | 2018-08-20 | 2021-05-11 | Amicus Therapeutics, Inc. | métodos de tratamento de doença de fabry em pacientes com uma mutação no gene gla |
WO2020046132A1 (en) | 2018-08-31 | 2020-03-05 | Leiden University | Pharmacological chaperones for enzyme treatment therapy |
NL2021840B1 (en) | 2018-10-19 | 2020-05-13 | Univ Leiden | Pharmacological Chaperones For Enzyme Treatment Therapy |
TW202042812A (zh) | 2019-01-22 | 2020-12-01 | 美商阿米庫斯醫療股份有限公司 | 減少法布瑞氏症患者中之腦血管事件之方法 |
JP2022536687A (ja) | 2019-06-11 | 2022-08-18 | アミカス セラピューティックス インコーポレイテッド | 腎機能障害を有する患者のファブリー病を治療する方法 |
TW202120087A (zh) * | 2019-08-07 | 2021-06-01 | 美商阿米庫斯醫療股份有限公司 | 治療在GLA基因中具有突變的患者的Fabry氏病之方法 |
EP4025242A1 (en) | 2019-09-06 | 2022-07-13 | Amicus Therapeutics, Inc. | Method for capturing and purification of biologics |
JP2023516753A (ja) * | 2020-03-06 | 2023-04-20 | アミカス セラピューティックス インコーポレイテッド | Gla遺伝子の突然変異を有する患者におけるファブリー病を治療する方法 |
US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
CA3225511A1 (en) | 2021-07-12 | 2023-01-19 | Franklin Johnson | Methods of treating fabry disease in pediatric patients |
WO2023215865A1 (en) | 2022-05-05 | 2023-11-09 | Amicus Therapeutics, Inc. | Methods for treating pompe disease |
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EP3432882B1 (en) | 2016-03-22 | 2021-09-01 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having the g9331a mutation in the gla gene |
US20190183869A1 (en) | 2016-07-19 | 2019-06-20 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naïve and ert-experienced patients |
JP2020503900A (ja) | 2017-01-10 | 2020-02-06 | アミカス セラピューティックス インコーポレイテッド | ファブリー病の処置のための組換えアルファ−ガラクトシダーゼa |
CA3065298A1 (en) | 2017-05-30 | 2018-12-06 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
SG11202000423XA (en) | 2017-07-19 | 2020-02-27 | Amicus Therapeutics Inc | Treatment of fabry disease in ert-naïve and ert-experienced patients |
HRP20221366T1 (hr) | 2017-08-28 | 2023-01-06 | Amicus Therapeutics, Inc. | Postupci poboljšanja i/ili stabilizacije srčane funkcije kod bolesnika s fabrijevom bolešću |
DK3749308T3 (da) | 2018-02-06 | 2024-01-22 | Amicus Therapeutics Inc | Behandling af patienter med klassisk fabrys sygdom med migalastat |
WO2019157047A1 (en) | 2018-02-06 | 2019-08-15 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
BR112021003137A2 (pt) | 2018-08-20 | 2021-05-11 | Amicus Therapeutics, Inc. | métodos de tratamento de doença de fabry em pacientes com uma mutação no gene gla |
JP2022536687A (ja) | 2019-06-11 | 2022-08-18 | アミカス セラピューティックス インコーポレイテッド | 腎機能障害を有する患者のファブリー病を治療する方法 |
TW202120087A (zh) | 2019-08-07 | 2021-06-01 | 美商阿米庫斯醫療股份有限公司 | 治療在GLA基因中具有突變的患者的Fabry氏病之方法 |
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2009
- 2009-02-12 DK DK09710178.6T patent/DK2252313T3/en active
- 2009-02-12 DK DK18201960.4T patent/DK3470077T3/da active
- 2009-02-12 DK DK15162630.6T patent/DK2946785T3/en active
- 2009-02-12 EP EP09710178.6A patent/EP2252313B1/en active Active
- 2009-02-12 ES ES15162630T patent/ES2716502T3/es active Active
- 2009-02-12 PT PT97101786T patent/PT2252313E/pt unknown
- 2009-02-12 PT PT15162630T patent/PT2946785T/pt unknown
- 2009-02-12 LT LTEP18201960.4T patent/LT3470077T/lt unknown
- 2009-02-12 EP EP15162630.6A patent/EP2946785B1/en active Active
- 2009-02-12 PL PL09710178T patent/PL2252313T3/pl unknown
- 2009-02-12 SI SI200931229T patent/SI2252313T1/sl unknown
- 2009-02-12 ES ES18201960T patent/ES2836121T3/es active Active
- 2009-02-12 PL PL15162630T patent/PL2946785T3/pl unknown
- 2009-02-12 WO PCT/US2009/033963 patent/WO2009102895A2/en active Application Filing
- 2009-02-12 SI SI200932099T patent/SI3470077T1/sl unknown
- 2009-02-12 EP EP20192637.5A patent/EP3824900A1/en active Pending
- 2009-02-12 CA CA2715407A patent/CA2715407C/en active Active
- 2009-02-12 AU AU2009214648A patent/AU2009214648B2/en active Active
- 2009-02-12 HU HUE15162630A patent/HUE042882T2/hu unknown
- 2009-02-12 JP JP2010546901A patent/JP5844045B2/ja active Active
- 2009-02-12 LT LTEP15162630.6T patent/LT2946785T/lt unknown
- 2009-02-12 PT PT182019604T patent/PT3470077T/pt unknown
- 2009-02-12 ES ES09710178.6T patent/ES2541933T3/es active Active
- 2009-02-12 EP EP18201960.4A patent/EP3470077B1/en active Active
- 2009-02-12 SI SI200931920T patent/SI2946785T1/sl unknown
- 2009-02-12 MX MX2010008835A patent/MX2010008835A/es active IP Right Grant
- 2009-02-12 PL PL18201960T patent/PL3470077T3/pl unknown
- 2009-02-12 HU HUE18201960A patent/HUE051377T2/hu unknown
- 2009-02-12 HU HUE09710178A patent/HUE026543T2/hu unknown
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2010
- 2010-08-12 US US12/855,468 patent/US8592362B2/en not_active Ceased
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2013
- 2013-10-15 US US14/054,369 patent/US9095584B2/en active Active
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2014
- 2014-09-10 AU AU2014221321A patent/AU2014221321B2/en active Active
- 2014-12-02 JP JP2014244283A patent/JP2015091239A/ja active Pending
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2015
- 2015-06-05 US US14/731,603 patent/US9545397B2/en active Active
- 2015-07-06 HR HRP20150728TT patent/HRP20150728T1/hr unknown
- 2015-07-08 CY CY20151100595T patent/CY1116466T1/el unknown
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2016
- 2016-03-01 JP JP2016039171A patent/JP6672013B2/ja active Active
- 2016-07-20 AU AU2016206297A patent/AU2016206297A1/en not_active Abandoned
- 2016-09-19 US US15/268,662 patent/US20170003301A1/en not_active Abandoned
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2017
- 2017-11-30 AU AU2017268649A patent/AU2017268649A1/en not_active Abandoned
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2018
- 2018-07-26 US US16/046,247 patent/US10813921B2/en active Active
- 2018-12-17 US US16/222,305 patent/USRE48608E1/en active Active
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2019
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2020
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2021
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