PL2946785T3 - Sposób przewidywania odpowiedzi na leczenie chorób farmakologicznymi białkami chaperonowymi - Google Patents

Sposób przewidywania odpowiedzi na leczenie chorób farmakologicznymi białkami chaperonowymi

Info

Publication number
PL2946785T3
PL2946785T3 PL15162630T PL15162630T PL2946785T3 PL 2946785 T3 PL2946785 T3 PL 2946785T3 PL 15162630 T PL15162630 T PL 15162630T PL 15162630 T PL15162630 T PL 15162630T PL 2946785 T3 PL2946785 T3 PL 2946785T3
Authority
PL
Poland
Prior art keywords
diseases
pharmacological chaperone
predict response
chaperone treatment
treatment
Prior art date
Application number
PL15162630T
Other languages
English (en)
Inventor
Benjamin Elfrida
Hung V Do
Xiaoyang Wu
John Flanagan
Brandon Wustman
Original Assignee
Amicus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=40957500&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PL2946785(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of PL2946785T3 publication Critical patent/PL2946785T3/pl

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING 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
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/34Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893Chemical 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
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/435Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
    • G01N2333/46Assays involving biological materials from specific organisms or of a specific nature from animals; from humans from vertebrates
    • G01N2333/47Assays involving proteins of known structure or function as defined in the subgroups
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90Enzymes; Proenzymes
    • G01N2333/914Hydrolases (3)
    • G01N2333/924Hydrolases (3) acting on glycosyl compounds (3.2)
    • G01N2333/94Hydrolases (3) acting on glycosyl compounds (3.2) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/04Endocrine or metabolic disorders
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/38Pediatrics
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/52Predicting 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)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Zoology (AREA)
  • Urology & Nephrology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & 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)
  • Cell Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Food Science & Technology (AREA)
  • General Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Endocrinology (AREA)
PL15162630T 2008-02-12 2009-02-12 Sposób przewidywania odpowiedzi na leczenie chorób farmakologicznymi białkami chaperonowymi PL2946785T3 (pl)

Applications Claiming Priority (7)

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
EP15162630.6A EP2946785B1 (en) 2008-02-12 2009-02-12 Method to predict response to pharmacological chaperone treatment of diseases

Publications (1)

Publication Number Publication Date
PL2946785T3 true PL2946785T3 (pl) 2019-04-30

Family

ID=40957500

Family Applications (3)

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
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

Family Applications Before (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

Family Applications After (1)

Application Number Title Priority Date Filing Date
PL18201960T PL3470077T3 (pl) 2008-02-12 2009-02-12 Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi

Country Status (16)

Country Link
US (8) US8592362B2 (pl)
EP (4) EP2946785B1 (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) HUE042882T2 (pl)
LT (2) LT3470077T (pl)
MX (1) MX2010008835A (pl)
PL (3) PL2252313T3 (pl)
PT (3) PT2946785T (pl)
SI (3) SI3470077T1 (pl)
WO (1) WO2009102895A2 (pl)

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE027569T2 (en) 2006-05-16 2016-10-28 Amicus Therapeutics Inc Preparation for the treatment of Fabry disease
PL2252313T3 (pl) 2008-02-12 2015-09-30 Amicus Therapeutics Inc Sposób przewidywania odpowiedzi na leczenie chorób farmakologicznymi białkami chaperonowymi
AU2009223125A1 (en) * 2008-03-12 2009-09-17 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for Pompe disease
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
PE20170906A1 (es) 2014-09-30 2017-07-12 Amicus Therapeutics Inc Alfa-glucosidasa acida muy potente con hidratos de carbono potenciados
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
MX394968B (es) 2015-12-30 2025-03-24 Amicus Therapeutics Inc Alfa-glucosidasa con mayor cantidad de acido para el tratamiento de la enfermedad de pompe.
RS62212B1 (sr) 2015-12-30 2021-09-30 Amicus Therapeutics Inc Augmentirana kiselina alfa-glukozidaza za lečenje pompeove bolesti
JP7098529B2 (ja) * 2016-03-22 2022-07-11 アミカス セラピューティックス インコーポレイテッド Gla遺伝子にg9331a変異を有する患者においてファブリー病を処置する方法
WO2017173060A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
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
MX2018011951A (es) * 2016-03-30 2019-02-13 Amicus Therapeutics Inc Metodo para seleccionar proteinas recombinantes ricas en m6p.
US12070453B2 (en) 2016-07-19 2024-08-27 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
IL266047B2 (en) * 2016-10-20 2025-02-01 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
AU2018270925B2 (en) 2017-05-15 2025-02-20 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
PT3630114T (pt) 2017-05-30 2024-02-01 Amicus Therapeutics Inc Migalastato para tratamento de pacientes com fabry com deficiência renal
CA3065298C (en) 2017-05-30 2025-05-06 Amicus Therapeutics, Inc. Methods of treating 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
ES2931054T3 (es) 2017-08-28 2022-12-23 Amicus Therapeutics Inc Métodos para potenciar y/o estabilizar la función cardíaca en pacientes con enfermedad de Fabry
EP4717319A2 (en) 2018-02-06 2026-04-01 Amicus Therapeutics, Inc. Methods of treating fabry disease in pregnant patients
PT3749308T (pt) 2018-02-06 2024-01-25 Amicus Therapeutics Inc Tratamento de pacientes com doença de fabry clássica com migalastato
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
SG11202101117QA (en) 2018-08-20 2021-03-30 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
WO2020252129A1 (en) 2019-06-11 2020-12-17 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having renal impairment
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
CA3149955A1 (en) 2019-09-06 2021-03-11 Kumar DHANASEKHARAN Method for capturing and purification of biologics
SI4114390T1 (sl) * 2020-03-06 2025-03-31 Amicus Therapeutics, Inc. Postopki zdravljenja Fabryjeve bolezni pri bolnikih z mutacijo gena GLA
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
US20240293387A1 (en) 2021-07-12 2024-09-05 Amicus Therapeutics, Inc. Methods of Treating Fabry Disease in Pediatric Patients
US20260041744A1 (en) 2022-05-05 2026-02-12 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
JP2025540135A (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 小児患者の乳児発症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

Family Cites Families (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US21381A (en) * 1858-08-31 Bracelet
US270486A (en) * 1883-01-09 Bundle-separating attachment for self-binding harvesters
US203019A (en) * 1878-04-30 Improvement in circuits for acoustic or telephonic telegraphs
US264467A (en) * 1882-09-19 Chables h
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
AU734905B2 (en) 1996-04-10 2001-06-28 Regents Of The University Of California, The 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
EP1896083B1 (en) * 2005-05-17 2016-03-02 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
WO2008045015A1 (en) 2005-06-08 2008-04-17 Amicus Therapeutics, Inc. Imino and amino sugar purification
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
HUE027569T2 (en) 2006-05-16 2016-10-28 Amicus Therapeutics Inc Preparation for the treatment of Fabry disease
DK1860101T3 (da) 2006-05-24 2011-07-25 Amicus Therapeutics Inc Tartrat salt af isofagomin og fremgangsmåder til anvendelse deraf
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
JP2010525084A (ja) 2007-04-26 2010-07-22 アミカス セラピューティックス インコーポレイテッド 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画
PL2252313T3 (pl) 2008-02-12 2015-09-30 Amicus Therapeutics Inc Sposób przewidywania odpowiedzi na leczenie chorób farmakologicznymi białkami chaperonowymi
WO2010048532A1 (en) 2008-10-24 2010-04-29 Amicus Therapeutics, Inc. Multiple compartment dosing model
US20110070643A1 (en) 2009-05-26 2011-03-24 Do Hung V Utilization of Pharmacological Chaperones to Improve Manufacturing and Purification of Biologics
US9206457B2 (en) 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
CA2781277C (en) 2009-11-17 2018-01-09 Baylor Research Institute Urinary triaosylceramide (gb3) as a marker of cardiac disease
MX2013010446A (es) 2011-03-11 2014-03-27 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de la enfermedad de fabry.
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
CA2917995C (en) 2012-07-17 2021-01-26 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
JP7098529B2 (ja) 2016-03-22 2022-07-11 アミカス セラピューティックス インコーポレイテッド Gla遺伝子にg9331a変異を有する患者においてファブリー病を処置する方法
US12070453B2 (en) 2016-07-19 2024-08-27 Amicus Therapeutics, Inc. Treatment of Fabry disease in ERT-naïve and ERT-experienced patients
WO2018132471A1 (en) 2017-01-10 2018-07-19 Amicus Therapeutics, Inc. Recombinant alpha-galactosidase a for treatment of fabry disease
CA3065298C (en) 2017-05-30 2025-05-06 Amicus Therapeutics, Inc. Methods of treating 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
ES2931054T3 (es) 2017-08-28 2022-12-23 Amicus Therapeutics Inc Métodos para potenciar y/o estabilizar la función cardíaca en pacientes con enfermedad de Fabry
EP4717319A2 (en) 2018-02-06 2026-04-01 Amicus Therapeutics, Inc. Methods of treating fabry disease in pregnant patients
PT3749308T (pt) 2018-02-06 2024-01-25 Amicus Therapeutics Inc Tratamento de pacientes com doença de fabry clássica com migalastato
SG11202101117QA (en) 2018-08-20 2021-03-30 Amicus Therapeutics Inc Methods of treating fabry disease in patients having a mutation in the gla gene
WO2020252129A1 (en) 2019-06-11 2020-12-17 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having renal impairment
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

Also Published As

Publication number Publication date
AU2014221321A1 (en) 2014-10-02
AU2014221321B2 (en) 2016-05-12
EP3470077A1 (en) 2019-04-17
EP2252313B1 (en) 2015-04-08
US20140206721A1 (en) 2014-07-24
WO2009102895A3 (en) 2009-12-23
AU2017268649A1 (en) 2018-01-04
AU2009214648B2 (en) 2014-11-13
DK2252313T3 (en) 2015-07-13
EP2252313A2 (en) 2010-11-24
US20250082621A9 (en) 2025-03-13
PL3470077T3 (pl) 2021-04-06
EP2252313A4 (en) 2011-04-13
AU2009214648A1 (en) 2009-08-20
EP3824900A1 (en) 2021-05-26
CA2715407A1 (en) 2009-08-20
PT3470077T (pt) 2020-11-30
DK3470077T3 (da) 2020-11-30
EP3470077B1 (en) 2020-08-26
DK2946785T3 (en) 2019-02-18
CY1116466T1 (el) 2017-03-15
US9095584B2 (en) 2015-08-04
EP2946785B1 (en) 2018-10-24
AU2016206297A1 (en) 2016-08-04
USRE48608E1 (en) 2021-06-29
PL2252313T3 (pl) 2015-09-30
HUE026543T2 (hu) 2016-06-28
JP2015091239A (ja) 2015-05-14
US8592362B2 (en) 2013-11-26
JP2021097673A (ja) 2021-07-01
US20150342940A1 (en) 2015-12-03
ES2541933T3 (es) 2015-07-28
EP2946785A1 (en) 2015-11-25
JP2011514152A (ja) 2011-05-06
AU2021218172A1 (en) 2021-10-07
ES2716502T3 (es) 2019-06-12
ES2836121T3 (es) 2021-06-24
PT2946785T (pt) 2019-02-01
HRP20201827T1 (hr) 2021-01-08
HRP20190143T1 (hr) 2019-03-22
AU2024219997A1 (en) 2024-10-10
CY1121386T1 (el) 2020-05-29
HUE042882T2 (hu) 2019-07-29
JP6672013B2 (ja) 2020-03-25
JP7277493B2 (ja) 2023-05-19
CY1123816T1 (el) 2022-03-24
US10813921B2 (en) 2020-10-27
JP2016163571A (ja) 2016-09-08
WO2009102895A2 (en) 2009-08-20
US20110152319A1 (en) 2011-06-23
LT3470077T (lt) 2021-02-25
AU2019219727A1 (en) 2019-09-12
PT2252313E (pt) 2015-08-26
HRP20150728T1 (hr) 2015-08-14
US20190000818A1 (en) 2019-01-03
LT2946785T (lt) 2019-02-11
US9545397B2 (en) 2017-01-17
US20110212996A1 (en) 2011-09-01
JP2023109807A (ja) 2023-08-08
AU2019219727B2 (en) 2021-05-20
SI3470077T1 (sl) 2020-12-31
MX2010008835A (es) 2010-10-20
JP5844045B2 (ja) 2016-01-13
JP2025148351A (ja) 2025-10-07
JP2019088289A (ja) 2019-06-13
HUE051377T2 (hu) 2021-03-01
SI2946785T1 (sl) 2019-02-28
CA2715407C (en) 2022-07-26
US20210251971A1 (en) 2021-08-19
US20170003301A1 (en) 2017-01-05
SI2252313T1 (sl) 2015-08-31
JP6837469B2 (ja) 2021-03-03

Similar Documents

Publication Publication Date Title
HRP20190143T1 (hr) Postupak za predviđanje odgovora na liječenje bolesti farmakološkim šaperonom
IL219051A0 (en) Methods to predict clinical outcome of cancer
IL209855A0 (en) Supression of neuroedocrine diseases
EP2496176A4 (en) ENTZELLULARISIERUNGSVERFAHREN
PT2435825E (pt) Métodos para o tratamento de doenças
ZA201109278B (en) Method for treatment of diseases
EP2517095A4 (en) ABOUT STORAGE HANDLING
EP2464376A4 (en) METHOD FOR REPAIRING NERVE DENDING
PT2504698T (pt) Método para prever a resposta de uma doença oncológica a uma medida terapêutica
FR2945911B1 (fr) Procede de traitement contre l'ambroisie