SI2252313T1 - Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem - Google Patents
Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcemInfo
- Publication number
- SI2252313T1 SI2252313T1 SI200931229T SI200931229T SI2252313T1 SI 2252313 T1 SI2252313 T1 SI 2252313T1 SI 200931229 T SI200931229 T SI 200931229T SI 200931229 T SI200931229 T SI 200931229T SI 2252313 T1 SI2252313 T1 SI 2252313T1
- Authority
- SI
- Slovenia
- Prior art keywords
- diseases
- pharmacological chaperone
- predict response
- chaperone treatment
- treatment
- Prior art date
Links
- 108010006519 Molecular Chaperones Proteins 0.000 title 1
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 230000000144 pharmacologic effect Effects 0.000 title 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
-
- 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)
- Physics & Mathematics (AREA)
- Analytical Chemistry (AREA)
- Urology & Nephrology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biophysics (AREA)
- Genetics & Genomics (AREA)
- Obesity (AREA)
- Epidemiology (AREA)
- Food Science & Technology (AREA)
- Pathology (AREA)
- General Engineering & Computer Science (AREA)
- Cell Biology (AREA)
- General Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Emergency Medicine (AREA)
Applications Claiming Priority (6)
| 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 |
| EP09710178.6A EP2252313B1 (en) | 2008-02-12 | 2009-02-12 | Method to predict response to pharmacological chaperone treatment of diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SI2252313T1 true SI2252313T1 (sl) | 2015-08-31 |
Family
ID=40957500
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SI200931229T SI2252313T1 (sl) | 2008-02-12 | 2009-02-12 | Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem |
| SI200931920T SI2946785T1 (sl) | 2008-02-12 | 2009-02-12 | Postopek napovedovanja odziva na farmakološko zdravljenje bolezni s šaperoni |
| SI200932099T SI3470077T1 (sl) | 2008-02-12 | 2009-02-12 | Postopek za napovedovanje odgovora na zdravljenje bolezni s farmakološkim spremljevalcem |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SI200931920T SI2946785T1 (sl) | 2008-02-12 | 2009-02-12 | Postopek napovedovanja odziva na farmakološko zdravljenje bolezni s šaperoni |
| SI200932099T SI3470077T1 (sl) | 2008-02-12 | 2009-02-12 | Postopek za napovedovanje odgovora na zdravljenje bolezni s farmakološkim spremljevalcem |
Country Status (16)
| Country | Link |
|---|---|
| US (8) | US8592362B2 (enExample) |
| EP (4) | EP2946785B1 (enExample) |
| JP (7) | JP5844045B2 (enExample) |
| AU (7) | AU2009214648B2 (enExample) |
| CA (1) | CA2715407C (enExample) |
| CY (3) | CY1116466T1 (enExample) |
| DK (3) | DK2252313T3 (enExample) |
| ES (3) | ES2716502T3 (enExample) |
| HR (3) | HRP20150728T1 (enExample) |
| HU (3) | HUE051377T2 (enExample) |
| LT (2) | LT2946785T (enExample) |
| MX (1) | MX2010008835A (enExample) |
| PL (3) | PL2252313T3 (enExample) |
| PT (3) | PT2252313E (enExample) |
| SI (3) | SI2252313T1 (enExample) |
| WO (1) | WO2009102895A2 (enExample) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL2533050T6 (pl) | 2006-05-16 | 2016-05-31 | Amicus Therapeutics Inc | Możliwości leczenia choroby Fabry'ego |
| WO2009102895A2 (en) | 2008-02-12 | 2009-08-20 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| JP2011517556A (ja) * | 2008-03-12 | 2011-06-16 | アミカス セラピューティックス インコーポレイテッド | ポンペ病のための治療選択肢を診断し評価するための検定 |
| WO2011041718A2 (en) * | 2009-10-01 | 2011-04-07 | 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 |
| HUE064629T2 (hu) | 2014-09-30 | 2024-04-28 | Amicus Therapeutics Inc | Nagy potenciálú savas alfa-glükozidáz fokozott szénhidrátokkal |
| CA2979695C (en) | 2015-03-19 | 2025-02-06 | Translate Bio, Inc. | RNA THERAPY FOR POMPE DISEASE |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| SG11201804965SA (en) | 2015-12-30 | 2018-07-30 | Amicus Therapeutics Inc | Augmented acid alpha-glucosidase for the treatment of pompe disease |
| SMT202300412T1 (it) | 2015-12-30 | 2024-01-10 | Amicus Therapeutics Inc | Alfa-glucosidasi acida potenziata per il trattamento della malattia di pompe |
| KR20190087673A (ko) | 2016-03-22 | 2019-07-24 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법 |
| CN109475607B (zh) | 2016-03-30 | 2022-04-26 | 阿米库斯治疗学公司 | 包含重组酸性α-葡糖苷酶的配制品 |
| KR102343162B1 (ko) | 2016-03-30 | 2021-12-23 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 고 m6p 재조합 단백질의 선택 방법 |
| KR20250017756A (ko) | 2016-03-30 | 2025-02-04 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 재조합 산 알파-글루코시다제를 포함하는 제형 |
| CN109661230A (zh) | 2016-07-19 | 2019-04-19 | 阿米库斯治疗学公司 | 治疗ert初治的和已经历ert的患者中的法布里病 |
| 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 |
| ES3047063T3 (en) * | 2016-10-20 | 2025-12-03 | Sangamo Therapeutics Inc | Methods and compositions for the treatment of fabry disease |
| EA202290114A1 (ru) * | 2017-03-30 | 2022-03-29 | Амикус Терапьютикс, Инк. | Способ отбора рекомбинантных белков с высоким содержанием m6p |
| SI3624831T1 (sl) | 2017-05-15 | 2023-08-31 | Amicus Therapeutics, Inc. | Rekombinantna humana kisla alfa-glukozidaza |
| SI3630114T1 (sl) * | 2017-05-30 | 2024-03-29 | Amicus Therapeutics, Inc. | Migalastat za zdravljenje bolnikov s fabryevo boleznijo, z ledvično okvaro |
| CA3224546A1 (en) | 2017-05-30 | 2018-12-06 | Jeff Castelli | Use of migalastat in the treatment of fabry patients having renal impairment |
| WO2019017938A1 (en) | 2017-07-19 | 2019-01-24 | Amicus Therapeutics, Inc. | TREATMENT OF FABRY'S DISEASE IN PATIENTS WITHOUT SUBSTITUTED ENZYMATIC THERAPY (TES) AND PATIENTS SUBJECT TO TES |
| US20200222377A1 (en) | 2017-08-28 | 2020-07-16 | Amicus Therapeutics, Inc. | Methods Of Enhancing And/Or Stabilizing Cardiac Function In Patients With Fabry Disease |
| EP3749308B1 (en) | 2018-02-06 | 2023-10-25 | Amicus Therapeutics, Inc. | Treatment of patients with classic fabry disease with migalastat |
| CA3090496C (en) | 2018-02-06 | 2024-03-26 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| 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 |
| DK3840753T3 (da) | 2018-08-20 | 2024-10-28 | Amicus Therapeutics Inc | Migalastat til anvendelse i fremgangsmåder til behandling af fabrys sygdom hos patienter, der har en mutation i gla-genet |
| NL2021840B1 (en) | 2018-10-19 | 2020-05-13 | Univ Leiden | Pharmacological Chaperones For Enzyme Treatment Therapy |
| WO2020046132A1 (en) | 2018-08-31 | 2020-03-05 | Leiden University | 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 |
| AR120055A1 (es) | 2019-06-11 | 2022-02-02 | Amicus Therapeutics Inc | Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal |
| US11833164B2 (en) * | 2019-08-07 | 2023-12-05 | Amicus Therapeutics, Inc. | Methods of treating Fabry disease in patients having a mutation in the GLA gene |
| BR112022004000A2 (pt) | 2019-09-06 | 2022-05-31 | Amicus Therapeutics Inc | Método para captura e purificação de biológicas |
| HUE070116T2 (hu) * | 2020-03-06 | 2025-05-28 | Amicus Therapeutics Inc | Eljárások Fabry-kór kezelésére olyan betegekben, akiknek mutációja van a GLA génben |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
| KR20240034203A (ko) | 2021-07-12 | 2024-03-13 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 소아청소년 환자에서 파브리병을 치료하는 방법 |
| TW202400212A (zh) | 2022-05-05 | 2024-01-01 | 美商阿米庫斯醫療股份有限公司 | 用於治療龐貝氏病之方法 |
| 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 |
| KR20250110931A (ko) | 2022-12-02 | 2025-07-21 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 소아 환자의 영아-발병 폼페병 치료에 대한 방법 |
| 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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| KR20190087673A (ko) | 2016-03-22 | 2019-07-24 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법 |
| CN109661230A (zh) | 2016-07-19 | 2019-04-19 | 阿米库斯治疗学公司 | 治疗ert初治的和已经历ert的患者中的法布里病 |
| EP3568152A1 (en) | 2017-01-10 | 2019-11-20 | Amicus Therapeutics, Inc. | Recombinant alpha-galactosidase a for treatment of fabry disease |
| CA3224546A1 (en) | 2017-05-30 | 2018-12-06 | Jeff Castelli | Use of migalastat in the treatment of fabry patients having renal impairment |
| WO2019017938A1 (en) | 2017-07-19 | 2019-01-24 | Amicus Therapeutics, Inc. | TREATMENT OF FABRY'S DISEASE IN PATIENTS WITHOUT SUBSTITUTED ENZYMATIC THERAPY (TES) AND PATIENTS SUBJECT TO TES |
| US20200222377A1 (en) | 2017-08-28 | 2020-07-16 | Amicus Therapeutics, Inc. | Methods Of Enhancing And/Or Stabilizing Cardiac Function In Patients With Fabry Disease |
| CA3090496C (en) | 2018-02-06 | 2024-03-26 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| EP3749308B1 (en) | 2018-02-06 | 2023-10-25 | Amicus Therapeutics, Inc. | Treatment of patients with classic fabry disease with migalastat |
| DK3840753T3 (da) | 2018-08-20 | 2024-10-28 | Amicus Therapeutics Inc | Migalastat til anvendelse i fremgangsmåder til behandling af fabrys sygdom hos patienter, der har en mutation i gla-genet |
| AR120055A1 (es) | 2019-06-11 | 2022-02-02 | Amicus Therapeutics Inc | Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal |
| US11833164B2 (en) | 2019-08-07 | 2023-12-05 | Amicus Therapeutics, Inc. | Methods of treating Fabry disease in patients having a mutation in the GLA gene |
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