AU2009214648B2 - Method to predict response to pharmacological chaperone treatment of diseases - Google Patents

Method to predict response to pharmacological chaperone treatment of diseases Download PDF

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Publication number
AU2009214648B2
AU2009214648B2 AU2009214648A AU2009214648A AU2009214648B2 AU 2009214648 B2 AU2009214648 B2 AU 2009214648B2 AU 2009214648 A AU2009214648 A AU 2009214648A AU 2009214648 A AU2009214648 A AU 2009214648A AU 2009214648 B2 AU2009214648 B2 AU 2009214648B2
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gal
protein
gaa
activity
cells
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AU2009214648A1 (en
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Elfrida Benjamin
Hung V. Do
John Flanagan
Xiaoyang Wu
Brandon Wustman
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Amicus Therapeutics Inc
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Amicus Therapeutics Inc
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Priority to AU2014221321A priority Critical patent/AU2014221321B2/en
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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

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  • 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)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Zoology (AREA)
  • Analytical Chemistry (AREA)
  • Urology & Nephrology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Obesity (AREA)
  • Epidemiology (AREA)
  • Pathology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Physics & Mathematics (AREA)
  • Food Science & Technology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Emergency Medicine (AREA)
AU2009214648A 2008-02-12 2009-02-12 Method to predict response to pharmacological chaperone treatment of diseases Active AU2009214648B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2014221321A AU2014221321B2 (en) 2008-02-12 2014-09-10 Method to predict response to pharmacological chaperone treatment of diseases
AU2018220047A AU2018220047A1 (en) 2009-02-12 2018-08-22 A method for treatment of fabry disease

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US2814108P 2008-02-12 2008-02-12
US61/028,141 2008-02-12
US3568408P 2008-03-11 2008-03-11
US61/035,684 2008-03-11
US9363108P 2008-09-02 2008-09-02
US61/093,631 2008-09-02
US11349608P 2008-11-11 2008-11-11
US61/113,496 2008-11-11
PCT/US2009/033963 WO2009102895A2 (en) 2008-02-12 2009-02-12 Method to predict response to pharmacological chaperone treatment of diseases

Related Child Applications (2)

Application Number Title Priority Date Filing Date
AU2014221321A Division AU2014221321B2 (en) 2008-02-12 2014-09-10 Method to predict response to pharmacological chaperone treatment of diseases
AU2018220047A Addition AU2018220047A1 (en) 2009-02-12 2018-08-22 A method for treatment of fabry disease

Publications (2)

Publication Number Publication Date
AU2009214648A1 AU2009214648A1 (en) 2009-08-20
AU2009214648B2 true AU2009214648B2 (en) 2014-11-13

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Application Number Title Priority Date Filing Date
AU2009214648A Active AU2009214648B2 (en) 2008-02-12 2009-02-12 Method to predict response to pharmacological chaperone treatment of diseases
AU2014221321A Active AU2014221321B2 (en) 2008-02-12 2014-09-10 Method to predict response to pharmacological chaperone treatment of diseases
AU2016206297A Abandoned AU2016206297A1 (en) 2008-02-12 2016-07-20 Method to predict response to pharmacological chaperone treatment of diseases
AU2017268649A Abandoned AU2017268649A1 (en) 2008-02-12 2017-11-30 Method to predict response to pharmacological chaperone treatment of diseases
AU2019219727A Active AU2019219727B2 (en) 2008-02-12 2019-08-20 Method to predict response to pharmacological chaperone treatment of diseases
AU2021218172A Abandoned AU2021218172A1 (en) 2008-02-12 2021-08-20 Method to predict response to pharmacological chaperone treatment of diseases
AU2024219997A Pending AU2024219997A1 (en) 2008-02-12 2024-09-24 Method to predict response to pharmacological chaperone treatment of diseases

Family Applications After (6)

Application Number Title Priority Date Filing Date
AU2014221321A Active AU2014221321B2 (en) 2008-02-12 2014-09-10 Method to predict response to pharmacological chaperone treatment of diseases
AU2016206297A Abandoned AU2016206297A1 (en) 2008-02-12 2016-07-20 Method to predict response to pharmacological chaperone treatment of diseases
AU2017268649A Abandoned AU2017268649A1 (en) 2008-02-12 2017-11-30 Method to predict response to pharmacological chaperone treatment of diseases
AU2019219727A Active AU2019219727B2 (en) 2008-02-12 2019-08-20 Method to predict response to pharmacological chaperone treatment of diseases
AU2021218172A Abandoned AU2021218172A1 (en) 2008-02-12 2021-08-20 Method to predict response to pharmacological chaperone treatment of diseases
AU2024219997A Pending AU2024219997A1 (en) 2008-02-12 2024-09-24 Method to predict response to pharmacological chaperone treatment of diseases

Country Status (16)

Country Link
US (8) US8592362B2 (https=)
EP (4) EP2946785B1 (https=)
JP (7) JP5844045B2 (https=)
AU (7) AU2009214648B2 (https=)
CA (1) CA2715407C (https=)
CY (3) CY1116466T1 (https=)
DK (3) DK3470077T3 (https=)
ES (3) ES2541933T3 (https=)
HR (3) HRP20150728T1 (https=)
HU (3) HUE042882T2 (https=)
LT (2) LT2946785T (https=)
MX (1) MX2010008835A (https=)
PL (3) PL2252313T3 (https=)
PT (3) PT2252313E (https=)
SI (3) SI2252313T1 (https=)
WO (1) WO2009102895A2 (https=)

Families Citing this family (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2533050T6 (pl) 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
EP2946785B1 (en) 2008-02-12 2018-10-24 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
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
CN114540327B (zh) 2014-09-30 2026-03-24 阿米库斯治疗学公司 具有增强的碳水化合物的高强度酸性α-葡糖苷酶
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
KR20250007693A (ko) 2015-12-30 2025-01-14 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
HRP20231465T1 (hr) 2015-12-30 2024-03-01 Amicus Therapeutics, Inc. Poboljšana kisela alfa-glukozidaza za liječenje pompeove bolesti
KR20190087673A (ko) * 2016-03-22 2019-07-24 아미쿠스 세라퓨틱스, 인코포레이티드 Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
SMT202200279T1 (it) 2016-03-30 2022-09-14 Amicus Therapeutics Inc Formulazioni comprendenti alfa-glucosidasi acida ricombinante
CA3019354A1 (en) * 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
AU2017239641A1 (en) 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
TW202408513A (zh) 2016-07-19 2024-03-01 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
KR102712926B1 (ko) * 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
EA039750B1 (ru) * 2017-03-30 2022-03-10 Амикус Терапьютикс, Инк. Способ отбора рекомбинантных белков с высоким содержанием m6p
SMT202300187T1 (it) 2017-05-15 2023-07-20 Amicus Therapeutics Inc Alfa-glucosidasi acida umana ricombinante
RS65230B1 (sr) 2017-05-30 2024-03-29 Amicus Therapeutics Inc Migalastat za lečenje pacijenata koji boluju od fabrijeve bolesti sa oštećenjem bubrega
CA3224546C (en) 2017-05-30 2026-02-24 Amicus Therapeutics, Inc. Use of migalastat in the treatment of fabry patients
SG11202000423XA (en) 2017-07-19 2020-02-27 Amicus Therapeutics Inc Treatment of fabry disease in ert-naïve and ert-experienced patients
US20200222377A1 (en) 2017-08-28 2020-07-16 Amicus Therapeutics, Inc. Methods Of Enhancing And/Or Stabilizing Cardiac Function In Patients With Fabry Disease
PT3749308T (pt) 2018-02-06 2024-01-25 Amicus Therapeutics Inc Tratamento de pacientes com doença de fabry clássica com migalastato
KR20200128676A (ko) 2018-02-06 2020-11-16 아미쿠스 세라퓨틱스, 인코포레이티드 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도
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
US12594268B2 (en) 2018-08-20 2026-04-07 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
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.
KR20220044560A (ko) * 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
EP4025242A1 (en) 2019-09-06 2022-07-13 Amicus Therapeutics, Inc. Method for capturing and purification of biologics
KR20220150902A (ko) 2020-02-10 2022-11-11 아미쿠스 세라퓨틱스, 인코포레이티드 파브리 질병을 치료하는 방법
AU2021230371A1 (en) * 2020-03-06 2022-09-29 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
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
EP4518885A1 (en) 2022-05-05 2025-03-12 Amicus Therapeutics, Inc. Methods for treating pompe disease
WO2024119091A1 (en) 2022-12-02 2024-06-06 Amicus Therapeutics, Inc. Fexamethods for treating infantile-onset pompe disease in pediatric patients
AR131256A1 (es) 2022-12-02 2025-03-05 Amicus Therapeutics Inc Métodos para tratar la enfermedad de pompe de inicio tardío en pacientes pediátricos
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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999062517A1 (en) * 1998-06-01 1999-12-09 Fan Jian Qiang METHOD OF ENHANCING LYSOSOMAL α-GALACTOSIDASE A
US20050203019A1 (en) * 2001-10-09 2005-09-15 Oregon Health & Science University Pharmacoperones for correcting disease states involving protein misfolding

Family Cites Families (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US264467A (en) * 1882-09-19 Chables h
US203019A (en) * 1878-04-30 Improvement in circuits for acoustic or telephonic telegraphs
US270486A (en) * 1883-01-09 Bundle-separating attachment for self-binding harvesters
US21381A (en) * 1858-08-31 Bracelet
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
US5900360A (en) 1996-04-10 1999-05-04 Welch; William J. Correction of genetic defects using chemical chaperones
US5948653A (en) 1997-03-21 1999-09-07 Pati; Sushma Sequence alterations using homologous recombination
US6244113B1 (en) 1999-10-29 2001-06-12 University Of Alabama In Huntsville Method and apparatus for measuring microgravity acceleration
EP3782655A1 (en) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
SI2027137T1 (sl) 2005-06-08 2015-06-30 Amicus Therapeutics, Inc. Čiščenje imino in amino sladkorja
AU2006254796B2 (en) * 2005-06-08 2012-08-16 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
PL2533050T6 (pl) * 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
PL1860101T3 (pl) 2006-05-24 2011-10-31 Amicus Therapeutics Inc Sól winianu izofagominy i sposoby zastosowania
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
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.
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
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
AU2010254092B2 (en) 2009-05-26 2015-11-12 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
WO2011063048A2 (en) 2009-11-17 2011-05-26 Baylor Research Institute Urinary triaosylceramide (gb3) as a marker of cardiac disease
SG10201604757RA (en) 2011-03-11 2016-08-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
WO2014014938A1 (en) 2012-07-17 2014-01-23 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
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
KR20190087673A (ko) 2016-03-22 2019-07-24 아미쿠스 세라퓨틱스, 인코포레이티드 Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
TW202408513A (zh) 2016-07-19 2024-03-01 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
US11357834B2 (en) 2017-01-10 2022-06-14 Amicus Therapeutics, Inc. Recombinant α-galactosidase A for treatment of Fabry disease
CA3224546C (en) 2017-05-30 2026-02-24 Amicus Therapeutics, Inc. Use of migalastat in the treatment of fabry patients
SG11202000423XA (en) 2017-07-19 2020-02-27 Amicus Therapeutics Inc Treatment of fabry disease in ert-naïve and ert-experienced patients
US20200222377A1 (en) 2017-08-28 2020-07-16 Amicus Therapeutics, Inc. Methods Of Enhancing And/Or Stabilizing Cardiac Function In Patients With Fabry Disease
KR20200128676A (ko) 2018-02-06 2020-11-16 아미쿠스 세라퓨틱스, 인코포레이티드 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도
PT3749308T (pt) 2018-02-06 2024-01-25 Amicus Therapeutics Inc Tratamento de pacientes com doença de fabry clássica com migalastato
US12594268B2 (en) 2018-08-20 2026-04-07 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
MX2021015352A (es) 2019-06-11 2022-04-06 Amicus Therapeutics Inc Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal.
KR20220044560A (ko) 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999062517A1 (en) * 1998-06-01 1999-12-09 Fan Jian Qiang METHOD OF ENHANCING LYSOSOMAL α-GALACTOSIDASE A
US20050203019A1 (en) * 2001-10-09 2005-09-15 Oregon Health & Science University Pharmacoperones for correcting disease states involving protein misfolding

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
Alfonso et al. "Miglustat (NB-DNJ) works as a chaperone for mutated acid beta-glycosidase in cells transfected with several Gaucher disease mutations" BLOOD CELLS, MOLECULES AND DISEASES.(2005);35(2):268-276 *
Fan J-Q et al. "Accelerated transport and maturation of lysosomal alpha-galactosidase A in Fabry Iymphoblasts by an enzyme inhibitor" NATURE MEDICINE,(1999); 5(1):112-115 *
Fan J-Q et al. "Cell-based screening of active-site specific chaperone for the treatment of Fabry disease", METHODS IN ENZYMOLOGY.(2003);363:412-420 *
Lei et al. "Enzyme enhancement activity of N-octyl-beta-valienamine on beta-glucosidase mutants associated with Gaucher disease", BIOCHIMICA ET BIOPHYSICA ACTA.(2007);1772(5):587-596 *
Shimotori Masaaki et al. "Novel mutations of the GLA gene in Japanese patients with Fabry disease and their functional characterization by active site specific chaperone" HUMAN MUTATION(2008)29(2):331 *
Wustman et al. "114. Pharmacological chaperone therapy for Gaucher disease: Mechanism of action, a survey of responsive mutations and phase I clinical trial results", MOLECULAR GENETICS AND METABOLISM.(2008);93(2):44. *

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