PL3470077T3 - Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi - Google Patents
Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymiInfo
- Publication number
- PL3470077T3 PL3470077T3 PL18201960T PL18201960T PL3470077T3 PL 3470077 T3 PL3470077 T3 PL 3470077T3 PL 18201960 T PL18201960 T PL 18201960T PL 18201960 T PL18201960 T PL 18201960T PL 3470077 T3 PL3470077 T3 PL 3470077T3
- Authority
- PL
- Poland
- 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)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Diabetes (AREA)
- Wood Science & Technology (AREA)
- Urology & Nephrology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Analytical Chemistry (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Biophysics (AREA)
- Genetics & Genomics (AREA)
- Obesity (AREA)
- Epidemiology (AREA)
- Pathology (AREA)
- General Physics & Mathematics (AREA)
- General Engineering & Computer Science (AREA)
- Food Science & Technology (AREA)
- Cell Biology (AREA)
- Gastroenterology & Hepatology (AREA)
- Endocrinology (AREA)
Applications Claiming Priority (8)
| 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 |
| EP15162630.6A EP2946785B1 (en) | 2008-02-12 | 2009-02-12 | Method to predict response to pharmacological chaperone treatment of diseases |
| EP18201960.4A EP3470077B1 (en) | 2008-02-12 | 2009-02-12 | Method to predict response to pharmacological chaperone treatment of diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL3470077T3 true PL3470077T3 (pl) | 2021-04-06 |
Family
ID=40957500
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL15162630T PL2946785T3 (pl) | 2008-02-12 | 2009-02-12 | Sposób przewidywania odpowiedzi na leczenie chorób farmakologicznymi białkami chaperonowymi |
| PL18201960T PL3470077T3 (pl) | 2008-02-12 | 2009-02-12 | Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi |
| PL09710178T PL2252313T3 (pl) | 2008-02-12 | 2009-02-12 | Sposób przewidywania odpowiedzi na leczenie chorób farmakologicznymi białkami chaperonowymi |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL15162630T PL2946785T3 (pl) | 2008-02-12 | 2009-02-12 | Sposób przewidywania odpowiedzi na leczenie chorób farmakologicznymi białkami chaperonowymi |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL09710178T PL2252313T3 (pl) | 2008-02-12 | 2009-02-12 | Sposób przewidywania odpowiedzi na leczenie chorób farmakologicznymi białkami chaperonowymi |
Country Status (16)
| Country | Link |
|---|---|
| US (8) | US8592362B2 (pl) |
| EP (4) | EP3470077B1 (pl) |
| JP (7) | JP5844045B2 (pl) |
| AU (7) | AU2009214648B2 (pl) |
| CA (1) | CA2715407C (pl) |
| CY (3) | CY1116466T1 (pl) |
| DK (3) | DK2252313T3 (pl) |
| ES (3) | ES2541933T3 (pl) |
| HR (3) | HRP20150728T1 (pl) |
| HU (3) | HUE026543T2 (pl) |
| LT (2) | LT3470077T (pl) |
| MX (1) | MX2010008835A (pl) |
| PL (3) | PL2946785T3 (pl) |
| PT (3) | PT2252313E (pl) |
| SI (3) | SI2252313T1 (pl) |
| WO (1) | WO2009102895A2 (pl) |
Families Citing this family (40)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2024745B1 (en) | 2006-05-16 | 2015-07-08 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for fabry disease |
| SI2252313T1 (sl) | 2008-02-12 | 2015-08-31 | Amicus Therapeutics, Inc. | Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem |
| CA2718059A1 (en) * | 2008-03-12 | 2009-09-17 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for pompe disease |
| 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 |
| MY186336A (en) | 2014-09-30 | 2021-07-13 | Amicus Therapeutics Inc | Highly potent acid alpha-glucosidase with enhanced carbohydrates |
| JP6895892B2 (ja) * | 2015-03-19 | 2021-06-30 | トランスレイト バイオ， インコーポレイテッド | ポンペ病のｍＲＮＡ治療 |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| PL3957320T3 (pl) | 2015-12-30 | 2024-02-19 | Amicus Therapeutics, Inc. | Wzmocniona kwaśna alfa-glukozydaza do leczenia choroby Pompego |
| CA3010205A1 (en) | 2015-12-30 | 2017-07-06 | Amicus Therapeutics, Inc. | Augmented acid alpha-glucosidase for the treatment of pompe disease |
| 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 |
| IL301319A (en) | 2016-03-30 | 2023-05-01 | Amicus Therapeutics Inc | Formulations that include recombinant acid alpha-glucosidase |
| SMT202200279T1 (it) | 2016-03-30 | 2022-09-14 | Amicus Therapeutics Inc | Formulazioni comprendenti alfa-glucosidasi acida ricombinante |
| MX2018011951A (es) * | 2016-03-30 | 2019-02-13 | Amicus Therapeutics Inc | Metodo para seleccionar proteinas recombinantes ricas en m6p. |
| TWI875676B (zh) | 2016-07-19 | 2025-03-11 | 美商阿米庫斯醫療股份有限公司 | 治療ert初治的和已經歷ert的患者中之法布瑞氏症 |
| 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 |
| MX2019004487A (es) * | 2016-10-20 | 2020-02-07 | Sangamo Therapeutics Inc | Metodos y composiciones para el tratamiento de la enfermedad de fabry. |
| 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 |
| KR20240017111A (ko) | 2017-05-30 | 2024-02-06 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 신장 손상을 갖는 파브리 환자를 치료하는 방법 |
| EP4374918B1 (en) | 2017-05-30 | 2025-07-16 | 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 |
| PL4176877T3 (pl) | 2017-08-28 | 2025-04-07 | Amicus Therapeutics, Inc. | Sposoby poprawy i/lub stabilizacji czynności serca u pacjentów z chorobą fabry'ego |
| WO2019157047A1 (en) | 2018-02-06 | 2019-08-15 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| LT3749308T (lt) | 2018-02-06 | 2024-02-12 | Amicus Therapeutics, Inc. | Klasikine fabri liga sergančių pacientų gydymas migalastatu |
| 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 |
| SI3840753T1 (sl) | 2018-08-20 | 2025-01-31 | Amicus Therapeutics, Inc. | Migalastat za uporabo v postopkih zdravljenja Fabryjeve bolezni pri bolnikih z mutacijo gena GLA |
| 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 |
| MX2021015352A (es) | 2019-06-11 | 2022-04-06 | Amicus Therapeutics Inc | Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal. |
| BR112022002202A2 (pt) | 2019-08-07 | 2022-06-14 | Amicus Therapeutics Inc | Métodos de tratar doença de fabry em pacientes tendo uma mutação no gene gla |
| CA3149955A1 (en) | 2019-09-06 | 2021-03-11 | Kumar DHANASEKHARAN | 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 |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
| EP4370120A1 (en) | 2021-07-12 | 2024-05-22 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in pediatric patients |
| WO2023215865A1 (en) | 2022-05-05 | 2023-11-09 | Amicus Therapeutics, Inc. | Methods for treating pompe disease |
| 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 |
| WO2024119091A1 (en) | 2022-12-02 | 2024-06-06 | Amicus Therapeutics, Inc. | Fexamethods for treating infantile-onset pompe disease in pediatric patients |
| US20240197706A1 (en) | 2022-12-13 | 2024-06-20 | Amicus Therapeutics, Inc. | Methods of improving the pharmacokinetics of migalastat |
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| US21381A (en) * | 1858-08-31 | Bracelet | ||
| US264467A (en) * | 1882-09-19 | Chables h | ||
| US203019A (en) * | 1878-04-30 | Improvement in circuits for acoustic or telephonic telegraphs | ||
| US3586908A (en) | 1969-02-28 | 1971-06-22 | Robert E Vosteen | Automatic potential control system for electrophotography apparatus |
| US5401650A (en) * | 1990-10-24 | 1995-03-28 | The Mount Sinai School Of Medicine Of The City University Of New York | Cloning and expression of biologically active α-galactosidase A |
| US5863903A (en) | 1994-03-09 | 1999-01-26 | Novo Nordisk A/S | Use of hydroxy alkyl piperidine and pyrrolidine compounds to treat diabetes |
| US5844102A (en) | 1994-07-07 | 1998-12-01 | University Of Maryland Baltimore County | Glycohydrolase inhibitors, their preparation and use thereof |
| US6270954B1 (en) | 1996-04-10 | 2001-08-07 | The Regents Of The University Of California | Correction of genetic defects using chemical chaperones |
| JP2000509971A (ja) | 1996-04-10 | 2000-08-08 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 化学的シャペローンを用いる遺伝的欠陥の修正 |
| US5948653A (en) | 1997-03-21 | 1999-09-07 | Pati; Sushma | Sequence alterations using homologous recombination |
| US6274597B1 (en) | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
| US6244113B1 (en) | 1999-10-29 | 2001-06-12 | University Of Alabama In Huntsville | Method and apparatus for measuring microgravity acceleration |
| US7842470B2 (en) * | 2001-10-09 | 2010-11-30 | Oregon Health & Science University | Method for pharmacoperones correction of GnRHR mutant protein misfolding |
| EP3441090A1 (en) * | 2005-05-17 | 2019-02-13 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
| PT2027137E (pt) | 2005-06-08 | 2015-06-09 | Amicus Therapeutics Inc | Purificação de imino e amino-açúcares |
| MX2007015602A (es) * | 2005-06-08 | 2008-02-21 | Amicus Therapeutics Inc | Tratamiento de trastornos del sistema nervioso central asociados con mutaciones en genes que codifican las enzimas lisosomicas. |
| WO2007089591A2 (en) * | 2006-01-27 | 2007-08-09 | The Board Of Trustees Of The Leland Stanford Junior University | Compositions and methods for high throughput screening of pharmacological chaperones |
| EP2024745B1 (en) | 2006-05-16 | 2015-07-08 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for fabry disease |
| MX2007006175A (es) | 2006-05-24 | 2009-02-16 | Amicus Therapeutics Inc | Sal de tartrato de isofagomina y metodos de uso. |
| MX2009011473A (es) | 2007-04-26 | 2010-01-18 | Amicus Therapeutics Inc | Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas. |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| SI2252313T1 (sl) | 2008-02-12 | 2015-08-31 | Amicus Therapeutics, Inc. | Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem |
| WO2010048532A1 (en) | 2008-10-24 | 2010-04-29 | Amicus Therapeutics, Inc. | Multiple compartment dosing model |
| US9206457B2 (en) | 2009-05-26 | 2015-12-08 | Amicus Therapeutics, Inc. | Utilization of pharmacological chaperones to improve manufacturing and purification of biologics |
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| EA031874B1 (ru) | 2011-03-11 | 2019-03-29 | Амикус Терапьютикс, Инк. | Способ лечения болезни фабри |
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| US10155027B2 (en) | 2012-07-17 | 2018-12-18 | Amicus Therapeutics, Inc. | Alpha-galactosidase A and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease |
| 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 |
| TWI875676B (zh) | 2016-07-19 | 2025-03-11 | 美商阿米庫斯醫療股份有限公司 | 治療ert初治的和已經歷ert的患者中之法布瑞氏症 |
| TW201829770A (zh) | 2017-01-10 | 2018-08-16 | 美商阿米庫斯醫療股份有限公司 | 用於治療fabry氏病之重組α-半乳糖苷酶A |
| KR20240017111A (ko) | 2017-05-30 | 2024-02-06 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 신장 손상을 갖는 파브리 환자를 치료하는 방법 |
| SG11202000423XA (en) | 2017-07-19 | 2020-02-27 | Amicus Therapeutics Inc | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| PL4176877T3 (pl) | 2017-08-28 | 2025-04-07 | Amicus Therapeutics, Inc. | Sposoby poprawy i/lub stabilizacji czynności serca u pacjentów z chorobą fabry'ego |
| LT3749308T (lt) | 2018-02-06 | 2024-02-12 | Amicus Therapeutics, Inc. | Klasikine fabri liga sergančių pacientų gydymas migalastatu |
| WO2019157047A1 (en) | 2018-02-06 | 2019-08-15 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| SI3840753T1 (sl) | 2018-08-20 | 2025-01-31 | Amicus Therapeutics, Inc. | Migalastat za uporabo v postopkih zdravljenja Fabryjeve bolezni pri bolnikih z mutacijo gena GLA |
| MX2021015352A (es) | 2019-06-11 | 2022-04-06 | Amicus Therapeutics Inc | Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal. |
| BR112022002202A2 (pt) | 2019-08-07 | 2022-06-14 | Amicus Therapeutics Inc | Métodos de tratar doença de fabry em pacientes tendo uma mutação no gene gla |
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