MX2010008835A - Metodo para predecir respuesta al tratamiento farmacologico con chaperona de enfermedades. - Google Patents
Metodo para predecir respuesta al tratamiento farmacologico con chaperona de enfermedades.Info
- Publication number
- MX2010008835A MX2010008835A MX2010008835A MX2010008835A MX2010008835A MX 2010008835 A MX2010008835 A MX 2010008835A MX 2010008835 A MX2010008835 A MX 2010008835A MX 2010008835 A MX2010008835 A MX 2010008835A MX 2010008835 A MX2010008835 A MX 2010008835A
- Authority
- MX
- Mexico
- Prior art keywords
- pharmacological chaperone
- diseases
- predict response
- treatment
- galactosidase
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 4
- 108010006519 Molecular Chaperones Proteins 0.000 title abstract 3
- 230000000144 pharmacologic effect Effects 0.000 title abstract 3
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 208000024720 Fabry Disease Diseases 0.000 abstract 2
- 102000002464 Galactosidases Human genes 0.000 abstract 1
- 108010093031 Galactosidases Proteins 0.000 abstract 1
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 238000000338 in vitro Methods 0.000 abstract 1
- LXBIFEVIBLOUGU-DPYQTVNSSA-N migalastat Chemical compound OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O LXBIFEVIBLOUGU-DPYQTVNSSA-N 0.000 abstract 1
- 229950007469 migalastat Drugs 0.000 abstract 1
- 230000004043 responsiveness Effects 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
- 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
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- 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
- G01N2800/04—Endocrine or metabolic disorders
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Hematology (AREA)
- Immunology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Diabetes (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Urology & Nephrology (AREA)
- Microbiology (AREA)
- Analytical Chemistry (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biophysics (AREA)
- Obesity (AREA)
- Epidemiology (AREA)
- Food Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Cell Biology (AREA)
- General Physics & Mathematics (AREA)
- Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Endocrinology (AREA)
Abstract
La presente invención proporciona métodos para determinar si un paciente con un trastorno por acumulación lisosomal se beneficiaría del tratamiento con una chaperona farmacológica específica. La presente invención ejemplifica un método in vitro para determinar la capacidad de respuesta de ~-galactosidasa A a una chaperona farmacológica tal como 1-desoxigalactonojirimicina en una línea celular que expresa una forma mutante de ~-galactosidasa A. la invención también proporciona un método para diagnosticar la enfermedad de Fabry en pacientes que se sospecha padecen la enfermedad de Fabry.
Applications Claiming Priority (5)
| 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 | |
| PCT/US2009/033963 WO2009102895A2 (en) | 2008-02-12 | 2009-02-12 | Method to predict response to pharmacological chaperone treatment of diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2010008835A true MX2010008835A (es) | 2010-10-20 |
Family
ID=40957500
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2010008835A MX2010008835A (es) | 2008-02-12 | 2009-02-12 | Metodo para predecir respuesta al tratamiento farmacologico con chaperona de enfermedades. |
Country Status (16)
| Country | Link |
|---|---|
| US (8) | US8592362B2 (es) |
| EP (4) | EP2946785B1 (es) |
| JP (7) | JP5844045B2 (es) |
| AU (7) | AU2009214648B2 (es) |
| CA (1) | CA2715407C (es) |
| CY (3) | CY1116466T1 (es) |
| DK (3) | DK3470077T3 (es) |
| ES (3) | ES2836121T3 (es) |
| HR (3) | HRP20150728T1 (es) |
| HU (3) | HUE026543T2 (es) |
| LT (2) | LT2946785T (es) |
| MX (1) | MX2010008835A (es) |
| PL (3) | PL3470077T3 (es) |
| PT (3) | PT3470077T (es) |
| SI (3) | SI2946785T1 (es) |
| WO (1) | WO2009102895A2 (es) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2009537149A (ja) | 2006-05-16 | 2009-10-29 | アミークス セラピューティックス インコーポレイテッド | ファブリー病に対する治療の選択を診断し、評価するためのアッセイ |
| PL3470077T3 (pl) | 2008-02-12 | 2021-04-06 | Amicus Therapeutics, Inc. | Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi |
| AU2009223125A1 (en) * | 2008-03-12 | 2009-09-17 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for Pompe disease |
| US20110112041A1 (en) * | 2009-10-01 | 2011-05-12 | Baylor Research Institute | 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 |
| ES2967620T3 (es) | 2014-09-30 | 2024-05-03 | Amicus Therapeutics Inc | Alfa-glucosidasa ácida altamente potente con carbohidratos potenciados |
| EP3270894B1 (en) * | 2015-03-19 | 2021-02-24 | Translate Bio, Inc. | Mrna therapy for pompe disease |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| HRP20210894T1 (hr) | 2015-12-30 | 2021-07-23 | Amicus Therapeutics, Inc. | Poboljšana kisela alfa-glukozidaza za liječenje pompeove bolesti |
| KR102510941B1 (ko) | 2015-12-30 | 2023-03-20 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
| CN109069494A (zh) * | 2016-03-22 | 2018-12-21 | 阿米库斯治疗学公司 | 治疗在gla基因中具有g9331a突变的患者的法布里病的方法 |
| KR102758570B1 (ko) | 2016-03-30 | 2025-01-23 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 재조합 산 알파-글루코시다제를 포함하는 제형 |
| PT3436053T (pt) | 2016-03-30 | 2022-07-08 | Amicus Therapeutics Inc | Formulações que compreendem a alfaglucosidase ácida recombinante |
| IL319770A (en) * | 2016-03-30 | 2025-05-01 | Amicus Therapeutics Inc | Method for selecting recombinant proteins with high mannose-6-phosphate content |
| EP3487502A1 (en) | 2016-07-19 | 2019-05-29 | 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 |
| JP7042263B2 (ja) * | 2016-10-20 | 2022-03-25 | サンガモ セラピューティクス， インコーポレイテッド | ファブリー病の治療のための方法および組成物 |
| EA039750B1 (ru) * | 2017-03-30 | 2022-03-10 | Амикус Терапьютикс, Инк. | Способ отбора рекомбинантных белков с высоким содержанием m6p |
| EP3624831B1 (en) | 2017-05-15 | 2023-03-29 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase |
| HUE065615T2 (hu) | 2017-05-30 | 2024-06-28 | Amicus Therapeutics Inc | Migalasztát, vesekárosodással szövõdött Fabry-kórban szenvedõ betegeg kezelésére |
| CA3224546A1 (en) | 2017-05-30 | 2018-12-06 | Jeff Castelli | Use of migalastat in the treatment of fabry patients having renal impairment |
| EA202090242A1 (ru) | 2017-07-19 | 2020-05-26 | Амикус Терапьютикс, Инк. | Лечение болезни фабри у фзт-наивных и фзт-леченных пациентов |
| EA202090564A1 (ru) | 2017-08-28 | 2020-06-15 | Амикус Терапьютикс, Инк. | Способы усиления и/или стабилизации сердечной функции у пациентов с болезнью фабри |
| RS65103B1 (sr) | 2018-02-06 | 2024-02-29 | Amicus Therapeutics Inc | Lečenje bolesnika sa klasičnom fabrijevom bolešću migalastatom |
| EP3749307B1 (en) | 2018-02-06 | 2025-12-10 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| US20220184185A1 (en) | 2018-07-25 | 2022-06-16 | Modernatx, Inc. | Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders |
| ES2993833T3 (en) | 2018-08-20 | 2025-01-10 | Amicus Therapeutics Inc | Migalastat for use in methods of treating fabry disease in patients having a mutation in the gla gene |
| 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 |
| BR112021024886A2 (pt) | 2019-06-11 | 2022-01-25 | Amicus Therapeutics Inc | Métodos de tratar a doença de fabry em pacientes com insuficiência renal |
| JP2021024864A (ja) * | 2019-08-07 | 2021-02-22 | 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遺伝子に突然変異を有する患者のファブリー病を治療する方法 |
| AU2020343680A1 (en) | 2019-09-06 | 2022-03-24 | Amicus Therapeutics, Inc. | Method for capturing and purification of biologics |
| HUE070116T2 (hu) * | 2020-03-06 | 2025-05-28 | Amicus Therapeutics Inc | Eljárások Fabry-kór kezelésére olyan betegekben, akiknek mutációja van a GLA génben |
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| US20240293387A1 (en) | 2021-07-12 | 2024-09-05 | Amicus Therapeutics, Inc. | Methods of Treating Fabry Disease in Pediatric Patients |
| JP2025515159A (ja) | 2022-05-05 | 2025-05-13 | アミカス セラピューティックス インコーポレイテッド | ポンペ病の治療方法 |
| TW202436620A (zh) | 2022-12-02 | 2024-09-16 | 美商阿米庫斯醫療股份有限公司 | 用於在兒科患者中治療嬰兒型龐貝氏症之方法 |
| JP2025540119A (ja) | 2022-12-02 | 2025-12-11 | アミカス セラピューティックス インコーポレイテッド | 小児患者の遅発型ポンペ病の治療方法 |
| US20240197706A1 (en) | 2022-12-13 | 2024-06-20 | Amicus Therapeutics, Inc. | Methods of improving the pharmacokinetics of migalastat |
| WO2026006625A1 (en) | 2024-06-26 | 2026-01-02 | Amicus Therapeutics, Inc. | Miglustat formulations comprising nitrosamine for use in the treatment of pompe disease |
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| CA3224546A1 (en) | 2017-05-30 | 2018-12-06 | Jeff Castelli | Use of migalastat in the treatment of fabry patients having renal impairment |
| EA202090242A1 (ru) | 2017-07-19 | 2020-05-26 | Амикус Терапьютикс, Инк. | Лечение болезни фабри у фзт-наивных и фзт-леченных пациентов |
| EA202090564A1 (ru) | 2017-08-28 | 2020-06-15 | Амикус Терапьютикс, Инк. | Способы усиления и/или стабилизации сердечной функции у пациентов с болезнью фабри |
| RS65103B1 (sr) | 2018-02-06 | 2024-02-29 | Amicus Therapeutics Inc | Lečenje bolesnika sa klasičnom fabrijevom bolešću migalastatom |
| EP3749307B1 (en) | 2018-02-06 | 2025-12-10 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| ES2993833T3 (en) | 2018-08-20 | 2025-01-10 | Amicus Therapeutics Inc | Migalastat for use in methods of treating fabry disease in patients having a mutation in the gla gene |
| BR112021024886A2 (pt) | 2019-06-11 | 2022-01-25 | Amicus Therapeutics Inc | Métodos de tratar a doença de fabry em pacientes com insuficiência renal |
| JP2021024864A (ja) | 2019-08-07 | 2021-02-22 | 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遺伝子に突然変異を有する患者のファブリー病を治療する方法 |
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