CY2016040I1 - Επιλογες θεραπειας για τη νοσο του fabry - Google Patents
Επιλογες θεραπειας για τη νοσο του fabryInfo
- Publication number
- CY2016040I1 CY2016040I1 CY2016040C CY2016040C CY2016040I1 CY 2016040 I1 CY2016040 I1 CY 2016040I1 CY 2016040 C CY2016040 C CY 2016040C CY 2016040 C CY2016040 C CY 2016040C CY 2016040 I1 CY2016040 I1 CY 2016040I1
- Authority
- CY
- Cyprus
- Prior art keywords
- fabry
- disease
- treatment options
- options
- treatment
- Prior art date
Links
- 208000024720 Fabry Disease Diseases 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
- 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
- A61K31/45—Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
- C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
- C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
- C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
- C07D211/40—Oxygen atoms
- C07D211/44—Oxygen atoms attached in position 4
- C07D211/46—Oxygen atoms attached in position 4 having a hydrogen atom as the second substituent in position 4
-
- 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
- G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
- G01N33/5094—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for blood cell populations
-
- 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)
-
- 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
- G01N2800/52—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)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Urology & Nephrology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Hematology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Cell Biology (AREA)
- Tropical Medicine & Parasitology (AREA)
- Pathology (AREA)
- General Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Ecology (AREA)
- Analytical Chemistry (AREA)
- Physics & Mathematics (AREA)
- Food Science & Technology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Physiology (AREA)
- Nutrition Science (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Vascular Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US80108906P | 2006-05-16 | 2006-05-16 | |
US85363106P | 2006-10-23 | 2006-10-23 | |
EP07797506.8A EP2024745B1 (en) | 2006-05-16 | 2007-05-16 | Assays for diagnosing and evaluating treatment options for fabry disease |
Publications (2)
Publication Number | Publication Date |
---|---|
CY2016040I2 CY2016040I2 (el) | 2017-06-28 |
CY2016040I1 true CY2016040I1 (el) | 2017-06-28 |
Family
ID=38723987
Family Applications (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20141100377T CY1115180T1 (el) | 2006-05-16 | 2014-05-27 | Επιλογες θεραπειας για τη νοσο του fabry |
CY20161100448T CY1117656T1 (el) | 2006-05-16 | 2016-05-24 | Επιλογες θεραπειας για τη νοσο του fabry |
CY2016040C CY2016040I1 (el) | 2006-05-16 | 2016-11-15 | Επιλογες θεραπειας για τη νοσο του fabry |
Family Applications Before (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20141100377T CY1115180T1 (el) | 2006-05-16 | 2014-05-27 | Επιλογες θεραπειας για τη νοσο του fabry |
CY20161100448T CY1117656T1 (el) | 2006-05-16 | 2016-05-24 | Επιλογες θεραπειας για τη νοσο του fabry |
Country Status (18)
Country | Link |
---|---|
US (9) | US7851143B2 (el) |
EP (5) | EP2024745B1 (el) |
JP (6) | JP2009537149A (el) |
AU (1) | AU2007253900A1 (el) |
CA (1) | CA2652553C (el) |
CY (3) | CY1115180T1 (el) |
DK (2) | DK2533050T6 (el) |
ES (3) | ES2573498T3 (el) |
HK (3) | HK1178975A1 (el) |
HU (2) | HUE027569T2 (el) |
LT (1) | LTC2787345I2 (el) |
LU (1) | LU93312I2 (el) |
MX (2) | MX351004B (el) |
NL (1) | NL300843I2 (el) |
PL (2) | PL2533050T6 (el) |
PT (1) | PT2533050E (el) |
SI (2) | SI2533050T1 (el) |
WO (1) | WO2007137072A2 (el) |
Families Citing this family (30)
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 |
AU2008245578A1 (en) * | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
DK2946785T3 (en) * | 2008-02-12 | 2019-02-18 | Amicus Therapeutics Inc | PROCEDURE FOR PREVENTING RESPONSE TO DISEASES OF PHARMACOLOGICAL CHAPERONE TREATMENT |
EP2260107A4 (en) * | 2008-03-12 | 2011-03-23 | Amicus Therapeutics Inc | TESTS FOR THE DIAGNOSIS AND ASSESSMENT OF TREATMENT OPTIONS FOR MORBUS POMPE |
JP5739898B2 (ja) * | 2009-11-17 | 2015-06-24 | ベイラー リサーチ インスティテュートBaylor Research Institute | 心疾患マーカーとしての尿中トリアオシルセラミド(gb3) |
CA2791994A1 (en) * | 2010-03-05 | 2011-09-09 | Baylor Research Institute | Involvement of androgen/androgen receptor pathway in fabry disease |
CA2829947C (en) * | 2011-03-11 | 2019-01-15 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of fabry disease |
HUE044867T2 (hu) | 2011-05-12 | 2019-11-28 | Proteostasis Therapeutics Inc | Proteosztázis szabályzók |
US20150258081A1 (en) * | 2011-12-22 | 2015-09-17 | Centogene Ip Gmbh | Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol |
AU2013202368B2 (en) | 2012-01-25 | 2016-06-16 | Proteostasis Therapeutics, Inc. | Proteasome activity modulating compounds |
WO2014127038A1 (en) * | 2013-02-12 | 2014-08-21 | Baylor Research Institute | Methods and compositions related to calcium homeostasis and fabry disease |
JP6546923B2 (ja) * | 2013-12-23 | 2019-07-17 | ビーシーエヌ ペプタイズ エセ.ア.Bcn Peptides S.A. | リソソーム蓄積障害または糖原病の処置における使用のためのエキソサイトーシス活性化化合物としてのビカルタミド類似体または(s)−ビカルタミド |
US9675627B2 (en) * | 2014-04-14 | 2017-06-13 | Amicus Therapeutics, Inc. | Dosing regimens for treating and/or preventing cerebral amyloidoses |
US9957494B2 (en) | 2014-07-25 | 2018-05-01 | Shire Human Genetic Therapies, Inc. | Crystal structure of human four-phosphate adaptor protein 2 glycolipid transfer protein like domain |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
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 |
US12020820B1 (en) | 2017-03-03 | 2024-06-25 | Cerner Innovation, Inc. | Predicting sphingolipidoses (fabry's disease) and decision support |
US11335461B1 (en) | 2017-03-06 | 2022-05-17 | Cerner Innovation, Inc. | Predicting glycogen storage diseases (Pompe disease) and decision support |
EP3630114B1 (en) * | 2017-05-30 | 2023-11-01 | Amicus Therapeutics, Inc. | Migalastat for treating fabry patients having renal impairment |
KR102631805B1 (ko) | 2017-05-30 | 2024-01-31 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 신장 손상을 갖는 파브리 환자를 치료하는 방법 |
US11923048B1 (en) | 2017-10-03 | 2024-03-05 | Cerner Innovation, Inc. | Determining mucopolysaccharidoses and decision support tool |
ES2969263T3 (es) * | 2018-02-06 | 2024-05-17 | Amicus Therapeutics Inc | Tratamiento de pacientes con enfermedad de Fabry clásica con migalastat |
AU2019217868A1 (en) | 2018-02-06 | 2020-10-01 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
WO2020006269A1 (en) | 2018-06-27 | 2020-01-02 | Proteostasis Therapeutics, Inc. | Proteasome activity enhancing compounds |
BR122023023216A2 (pt) | 2018-06-27 | 2024-02-20 | Proteostasis Therapeutics, Inc. | Compostos, composição farmacêutica e uso de uma quantidade eficaz de um composto |
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 |
KR20220044560A (ko) | 2019-08-07 | 2022-04-08 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법 |
US11191827B1 (en) | 2020-12-09 | 2021-12-07 | Tevogen Bio Inc. | COVID-19 peptide specific T-cells and methods of treating and preventing COVID-19 |
US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
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WO1990011353A1 (en) * | 1989-03-24 | 1990-10-04 | Research Corporation Technologies, Inc. | Recombinant alpha-galactosidase, a therapy for fabry disease |
CA2086413A1 (en) | 1990-06-29 | 1991-12-30 | Yohji Ezure | Piperidine derivatives |
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US7446098B2 (en) * | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
WO2004103368A1 (en) * | 2003-05-22 | 2004-12-02 | The Hospital For Sick Children | Treatment of tay sachs or sandhoff diseases by enhancing hexosaminidase activity |
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KR20080033242A (ko) | 2005-06-08 | 2008-04-16 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 리소좀 효소를 코딩하는 유전자의 돌연변이와 관련있는cns 질환의 치료 방법 |
US20100004156A1 (en) | 2005-07-27 | 2010-01-07 | Shalesh Kaushal | Small Compounds That Correct Protein Misfolding and Uses Thereof |
US7790364B2 (en) | 2006-01-27 | 2010-09-07 | 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 |
ATE500226T1 (de) | 2006-09-22 | 2011-03-15 | Krka Tovarna Zdravil D D Novo Mesto | Aripiprazolhemifumarat und verfahren zu dessen herstellung |
EP2142197A4 (en) | 2007-03-30 | 2010-11-10 | Amicus Therapeutics Inc | PROCESS FOR THE TREATMENT OF FABRY DISEASE USING PHARMACOLOGICAL CHAPERONS |
AU2008245578A1 (en) | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
DK2946785T3 (en) | 2008-02-12 | 2019-02-18 | Amicus Therapeutics Inc | PROCEDURE FOR PREVENTING RESPONSE TO DISEASES OF PHARMACOLOGICAL CHAPERONE TREATMENT |
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CA2829947C (en) | 2011-03-11 | 2019-01-15 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of fabry disease |
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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 |
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 |
EP3568152A1 (en) | 2017-01-10 | 2019-11-20 | Amicus Therapeutics, Inc. | Recombinant alpha-galactosidase a for treatment of fabry disease |
KR102631805B1 (ko) | 2017-05-30 | 2024-01-31 | 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 |
TWI845479B (zh) | 2017-08-28 | 2024-06-21 | 美商阿米庫斯醫療股份有限公司 | 增強及/或穩定Fabry氏症患者之心臟功能之方法 |
AU2019217868A1 (en) | 2018-02-06 | 2020-10-01 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
ES2969263T3 (es) | 2018-02-06 | 2024-05-17 | Amicus Therapeutics Inc | Tratamiento de pacientes con enfermedad de Fabry clásica con migalastat |
FI3840753T3 (fi) | 2018-08-20 | 2024-10-30 | Amicus Therapeutics Inc | Migalastaatti käytettäväksi fabryn taudin hoitomenetelmissä potilailla joilla on gla-geenin mutaatio |
WO2020252129A1 (en) | 2019-06-11 | 2020-12-17 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having renal impairment |
KR20220044560A (ko) | 2019-08-07 | 2022-04-08 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법 |
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