CY1121386T1 - Μεθοδος για την προβλεψη αποκρισης των ασθενειων σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης - Google Patents
Μεθοδος για την προβλεψη αποκρισης των ασθενειων σε φαρμακολογικη θεραπευτικη αγωγη σαπερονηςInfo
- Publication number
- CY1121386T1 CY1121386T1 CY20191100100T CY191100100T CY1121386T1 CY 1121386 T1 CY1121386 T1 CY 1121386T1 CY 20191100100 T CY20191100100 T CY 20191100100T CY 191100100 T CY191100100 T CY 191100100T CY 1121386 T1 CY1121386 T1 CY 1121386T1
- Authority
- CY
- Cyprus
- Prior art keywords
- saperone
- gal
- preventing disease
- pharmacological treatment
- disease response
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 3
- 230000004044 response Effects 0.000 title abstract 2
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 238000011458 pharmacological treatment Methods 0.000 title 1
- 101000718529 Saccharolobus solfataricus (strain ATCC 35092 / DSM 1617 / JCM 11322 / P2) Alpha-galactosidase Proteins 0.000 abstract 3
- NNISLDGFPWIBDF-MPRBLYSKSA-N alpha-D-Gal-(1->3)-beta-D-Gal-(1->4)-D-GlcNAc Chemical compound O[C@@H]1[C@@H](NC(=O)C)C(O)O[C@H](CO)[C@H]1O[C@H]1[C@H](O)[C@@H](O[C@@H]2[C@@H]([C@@H](O)[C@@H](O)[C@@H](CO)O2)O)[C@@H](O)[C@@H](CO)O1 NNISLDGFPWIBDF-MPRBLYSKSA-N 0.000 abstract 3
- 230000000144 pharmacologic effect Effects 0.000 abstract 3
- 150000002148 esters Chemical class 0.000 abstract 1
- 238000000338 in vitro Methods 0.000 abstract 1
- 230000035772 mutation Effects 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 230000004043 responsiveness Effects 0.000 abstract 1
- 150000003839 salts Chemical class 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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- 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
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- 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)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Diabetes (AREA)
- Physics & Mathematics (AREA)
- Analytical Chemistry (AREA)
- Wood Science & Technology (AREA)
- Urology & Nephrology (AREA)
- Microbiology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Genetics & Genomics (AREA)
- Biophysics (AREA)
- Obesity (AREA)
- Epidemiology (AREA)
- Pathology (AREA)
- General Physics & Mathematics (AREA)
- Food Science & Technology (AREA)
- Cell Biology (AREA)
- General Engineering & Computer Science (AREA)
- Gastroenterology & Hepatology (AREA)
- Endocrinology (AREA)
Abstract
Μια μέθοδος σύνταξης ενός πίνακα αναφοράς θεραπευτικής αντιμετώπισης, ο οποίος υποδεικνύει την αποκρισιμότητα μιας ή περισσοτέρων ειδικών μεταλλάξεων ενός α-Gal Α σε μια ειδική φαρμακολογική σαπερόνη, με την μέθοδο να περιλαμβάνει: την ανάλυση in vitro της απόκρισης στην ειδική φαρμακολογική σαπερόνη εντός ενός κυττάρου ξενιστή, το οποίο έχει μετασχηματισθεί με έναν φορέα νουκλεϊνικού οξέος, ο οποίος κωδικοποιεί έναν πρώτο μεταλλάκτη α-Gal Α και τη σύνταξη ενός καταλόγου, υπό μορφή πίνακα, αποκρίσιμων και μη-αποκρίσιμων μεταλλαγμένων μορφών ενός α-Gal Α. Η ειδική φαρμακολογική σαπερόνη είναι 1-δεοξυγαλακτονοζιριμυκίνη ή ένα φαρμακευτικό άλας ή εστέρας και το πρώτο κύτταρο είναι ένα ΗΕΚ-293 MSR κύτταρο.
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 | |
EP09710178.6A EP2252313B1 (en) | 2008-02-12 | 2009-02-12 | Method to predict response to pharmacological chaperone treatment of diseases |
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 |
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CY1121386T1 true CY1121386T1 (el) | 2020-05-29 |
Family
ID=40957500
Family Applications (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20151100595T CY1116466T1 (el) | 2008-02-12 | 2015-07-08 | Μεθοδος για την προβλεψη αποκρισης σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης των ασθενειων |
CY20191100100T CY1121386T1 (el) | 2008-02-12 | 2019-01-24 | Μεθοδος για την προβλεψη αποκρισης των ασθενειων σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης |
CY20201101114T CY1123816T1 (el) | 2008-02-12 | 2020-11-25 | Μεθοδος για την προβλεψη αποκρισης των ασθενειων σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20151100595T CY1116466T1 (el) | 2008-02-12 | 2015-07-08 | Μεθοδος για την προβλεψη αποκρισης σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης των ασθενειων |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20201101114T CY1123816T1 (el) | 2008-02-12 | 2020-11-25 | Μεθοδος για την προβλεψη αποκρισης των ασθενειων σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης |
Country Status (16)
Country | Link |
---|---|
US (8) | US8592362B2 (el) |
EP (4) | EP3470077B1 (el) |
JP (6) | JP5844045B2 (el) |
AU (6) | AU2009214648B2 (el) |
CA (1) | CA2715407C (el) |
CY (3) | CY1116466T1 (el) |
DK (3) | DK2252313T3 (el) |
ES (3) | ES2541933T3 (el) |
HR (3) | HRP20150728T1 (el) |
HU (3) | HUE051377T2 (el) |
LT (2) | LT3470077T (el) |
MX (1) | MX2010008835A (el) |
PL (3) | PL3470077T3 (el) |
PT (3) | PT3470077T (el) |
SI (3) | SI2252313T1 (el) |
WO (1) | WO2009102895A2 (el) |
Families Citing this family (40)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP2955520B1 (en) | 2006-05-16 | 2018-10-31 | Amicus Therapeutics, Inc. | Treatment options for fabry disease |
DK2252313T3 (en) | 2008-02-12 | 2015-07-13 | Amicus Therapeutics Inc | METHOD TO PREDICT response to Pharmacological chaperone TREATMENT OF DISEASES |
MX2010009875A (es) * | 2008-03-12 | 2010-11-26 | Amicus Therapeutics Inc | Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de pompe. |
AR078498A1 (es) * | 2009-10-01 | 2011-11-09 | Baylor Res Inst | Tratamiento de calvicie de patron masculino por induccion local del defecto metabolico de la enfermedad de fabry |
US20120283290A1 (en) * | 2011-05-06 | 2012-11-08 | Amicus Therapeutics Inc. | Quantitation of gl3 in urine |
FI3201320T3 (fi) | 2014-09-30 | 2024-01-08 | Amicus Therapeutics Inc | Erittäin tehokas hapan alfa-glukosidaasi parannetuilla hiilihydraateilla |
WO2016149508A1 (en) * | 2015-03-19 | 2016-09-22 | Shire Human Genetic Therapies, Inc. | Mrna therapy for pompe disease |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
PT3957320T (pt) | 2015-12-30 | 2023-11-23 | Amicus Therapeutics Inc | Alfa-glicosidase ácida aumentada para o tratamento da doença de pompe |
KR102510941B1 (ko) | 2015-12-30 | 2023-03-20 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
WO2017165164A1 (en) | 2016-03-22 | 2017-09-28 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having the g9331a mutation in the gla gene |
KR20220145918A (ko) | 2016-03-30 | 2022-10-31 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 고 m6p 재조합 단백질의 선택 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 |
CA3019128A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
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 |
CN110022904B (zh) * | 2016-10-20 | 2024-04-19 | 桑格摩生物治疗股份有限公司 | 用于治疗法布里病的方法和组合物 |
EA039750B1 (ru) * | 2017-03-30 | 2022-03-10 | Амикус Терапьютикс, Инк. | Способ отбора рекомбинантных белков с высоким содержанием m6p |
KR20200004414A (ko) | 2017-05-15 | 2020-01-13 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 재조합 인간 산 알파-글루코시다제 |
JP2020507562A (ja) | 2017-05-30 | 2020-03-12 | 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ファブリー患者の治療方法 |
EP3630114B1 (en) | 2017-05-30 | 2023-11-01 | Amicus Therapeutics, Inc. | Migalastat for 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 |
SG11202001707PA (en) | 2017-08-28 | 2020-03-30 | Amicus Therapeutics Inc | Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease |
FI3749308T3 (fi) | 2018-02-06 | 2024-01-18 | Amicus Therapeutics Inc | Potilaiden, joilla on klassinen fabryn tauti, hoito migalastaatilla |
EP3749307A1 (en) | 2018-02-06 | 2020-12-16 | 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 |
EP4289476A3 (en) | 2018-08-20 | 2024-02-21 | Amicus Therapeutics, Inc. | 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 |
TW202112372A (zh) | 2019-06-11 | 2021-04-01 | 美商阿米庫斯醫療股份有限公司 | 治療具有腎損害患者的Fabry氏病之方法 |
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 |
WO2021046443A1 (en) | 2019-09-06 | 2021-03-11 | Amicus Therapeutics, Inc. | Method for capturing and purification of biologics |
CN115427040A (zh) * | 2020-03-06 | 2022-12-02 | 阿米库斯治疗学公司 | 治疗在gla基因中具有突变的患者的法布里病的方法 |
US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
KR20240034203A (ko) | 2021-07-12 | 2024-03-13 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 소아청소년 환자에서 파브리병을 치료하는 방법 |
WO2023215865A1 (en) | 2022-05-05 | 2023-11-09 | Amicus Therapeutics, Inc. | Methods for treating pompe disease |
WO2024119070A1 (en) | 2022-12-02 | 2024-06-06 | Amicus Therapeutics, Inc. | Methods for treating late onset pompe disease in pediatric patients |
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 |
Family Cites Families (41)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US270486A (en) * | 1883-01-09 | Bundle-separating attachment for self-binding harvesters | ||
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 |
WO1997037645A1 (en) | 1996-04-10 | 1997-10-16 | The Regents Of The University Of California | Correction of genetic defects using chemical chaperones |
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 |
ES2572148T3 (es) * | 2005-05-17 | 2016-05-30 | Amicus Therapeutics Inc | Un método para el tratamiento de la enfermedad de Pompe usando 1-desoxinojirimicina y derivados |
ES2537089T3 (es) | 2005-06-08 | 2015-06-02 | Amicus Therapeutics, Inc. | Purificación de imino- y amino-azúcares |
CA2611011C (en) * | 2005-06-08 | 2014-01-28 | Amicus Therapeutics, Inc. | Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes |
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 |
EP2955520B1 (en) * | 2006-05-16 | 2018-10-31 | Amicus Therapeutics, Inc. | Treatment options for fabry disease |
DE602007013648D1 (de) | 2006-05-24 | 2011-05-19 | Amicus Therapeutics Inc | Tartratsalz aus Isofagomin und Verwendungsverfahren dafür |
US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
WO2008134628A2 (en) | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
DK2252313T3 (en) | 2008-02-12 | 2015-07-13 | Amicus Therapeutics Inc | METHOD TO PREDICT response to Pharmacological chaperone TREATMENT OF DISEASES |
US8321148B2 (en) | 2008-10-24 | 2012-11-27 | 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 |
US20110070643A1 (en) | 2009-05-26 | 2011-03-24 | Do Hung V | Utilization of Pharmacological Chaperones to Improve Manufacturing and Purification of Biologics |
TW201142297A (en) | 2009-11-17 | 2011-12-01 | Baylor Res Inst | Urinary triaosylceramide (Gb3) as a marker of cardiac disease |
US20140219986A1 (en) | 2011-03-11 | 2014-08-07 | Amicus Therapeutics ,Inc. | Dosing regimens for the treatment of fabry disease |
EP2874648A4 (en) | 2012-07-17 | 2015-12-30 | Amicus Therapeutics Inc | CO-FORMULATION OF ALPHA-GALACTOSIDASE A AND 1-DEOXYGALACTONOJIRIMYCIN |
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 |
WO2017165164A1 (en) | 2016-03-22 | 2017-09-28 | 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 |
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 |
JP2020507562A (ja) | 2017-05-30 | 2020-03-12 | 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ファブリー患者の治療方法 |
SG11202000423XA (en) | 2017-07-19 | 2020-02-27 | Amicus Therapeutics Inc | Treatment of fabry disease in ert-naïve and ert-experienced patients |
SG11202001707PA (en) | 2017-08-28 | 2020-03-30 | Amicus Therapeutics Inc | Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease |
EP3749307A1 (en) | 2018-02-06 | 2020-12-16 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
FI3749308T3 (fi) | 2018-02-06 | 2024-01-18 | Amicus Therapeutics Inc | Potilaiden, joilla on klassinen fabryn tauti, hoito migalastaatilla |
EP4289476A3 (en) | 2018-08-20 | 2024-02-21 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the gla gene |
TW202112372A (zh) | 2019-06-11 | 2021-04-01 | 美商阿米庫斯醫療股份有限公司 | 治療具有腎損害患者的Fabry氏病之方法 |
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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