HK1216230A1 - 個方法用作龐貝氏症的治療使用 -脫氧野尻黴素和衍生物 - Google Patents

個方法用作龐貝氏症的治療使用 -脫氧野尻黴素和衍生物

Info

Publication number
HK1216230A1
HK1216230A1 HK16104047.7A HK16104047A HK1216230A1 HK 1216230 A1 HK1216230 A1 HK 1216230A1 HK 16104047 A HK16104047 A HK 16104047A HK 1216230 A1 HK1216230 A1 HK 1216230A1
Authority
HK
Hong Kong
Prior art keywords
deoxynojirimycin
derivatives
treatment
pompe disease
pompe
Prior art date
Application number
HK16104047.7A
Other languages
English (en)
Inventor
Mugrage Benjamin
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
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of HK1216230A1 publication Critical patent/HK1216230A1/zh

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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
    • 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
    • A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/4525—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom
    • 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
    • A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/4545—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. pipamperone, anabasine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61K9/20—Pills, tablets, discs, rods
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/04—Drugs for disorders of the respiratory system for throat disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • 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
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D405/00—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
    • C07D405/02—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings
    • C07D405/06—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a carbon chain containing only aliphatic carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D409/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms
    • C07D409/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing two hetero rings
    • C07D409/06—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing two hetero rings linked by a carbon chain containing only aliphatic carbon atoms
HK16104047.7A 2005-05-17 2016-04-08 個方法用作龐貝氏症的治療使用 -脫氧野尻黴素和衍生物 HK1216230A1 (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US68224105P 2005-05-17 2005-05-17
US72932905P 2005-10-21 2005-10-21

Publications (1)

Publication Number Publication Date
HK1216230A1 true HK1216230A1 (zh) 2016-10-28

Family

ID=37432167

Family Applications (1)

Application Number Title Priority Date Filing Date
HK16104047.7A HK1216230A1 (zh) 2005-05-17 2016-04-08 個方法用作龐貝氏症的治療使用 -脫氧野尻黴素和衍生物

Country Status (12)

Country Link
US (3) US9181184B2 (zh)
EP (4) EP1896083B1 (zh)
JP (2) JP2008545657A (zh)
KR (1) KR20080025373A (zh)
AU (1) AU2006247065B8 (zh)
BR (1) BRPI0612928A2 (zh)
CA (1) CA2612538C (zh)
ES (2) ES2572148T3 (zh)
HK (1) HK1216230A1 (zh)
IL (3) IL187461A (zh)
MX (1) MX2007014470A (zh)
WO (1) WO2006125141A2 (zh)

Families Citing this family (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI2857036T1 (sl) * 2003-01-31 2017-05-31 Mount Sinai School Of Medicine Of New York University Kombinirana terapija za zdravljenje motenj pomanjkanja proteinov
CA2544086A1 (en) * 2003-10-29 2005-05-06 Macrozyme B.V. Use of a deoxynojirimycin derivative or a pharmaceutically salt thereof
EP1528056A1 (en) * 2003-10-29 2005-05-04 Academisch Ziekenhuis bij de Universiteit van Amsterdam Deoxynojirimycin analogues and their uses as glucosylceramidase inhibitors
EP1896083B1 (en) 2005-05-17 2016-03-02 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
PL2787345T3 (pl) 2006-05-16 2016-08-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry’ego
WO2007140184A2 (en) * 2006-05-24 2007-12-06 United Therapeutics Corporation Deoxynojirimycin and d-arabinitol analogs and methods of using
US8283180B2 (en) 2007-04-13 2012-10-09 Amicus Therapeutics, Inc. Periodic acid-schiff staining with detection in the infrared range
US9056101B2 (en) 2007-04-26 2015-06-16 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
PT2946785T (pt) 2008-02-12 2019-02-01 Amicus Therapeutics Inc Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas
CA2718059A1 (en) * 2008-03-12 2009-09-17 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for pompe disease
US20110189710A1 (en) * 2008-03-12 2011-08-04 Amicus Therapeutics, Inc. TREATMENT OF POMPE DISEASE WITH SPECIFIC PHARMACOLOGICAL CHAPERONES AND MONITORING TREATMENT USING SURROGATE MARkERS
WO2010015816A2 (en) * 2008-08-06 2010-02-11 Summit Corporation Plc Treatment of lysosomal storage disorders and other proteostatic diseases
US20100119502A1 (en) * 2008-11-11 2010-05-13 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
AT507745B1 (de) * 2008-12-16 2012-01-15 Forschungsholding Tu Graz Gmbh Fluorophile glykosidasehemmer
WO2010096369A1 (en) * 2009-02-18 2010-08-26 Amicus Therapeutics, Inc. Mouse model for pompe disease and methods of use thereof
WO2010096535A1 (en) * 2009-02-18 2010-08-26 Amicus Therapeutics, Inc. Method for diagnosing pompe disease
CN106420740A (zh) 2009-02-23 2017-02-22 伊莫根特病毒学有限责任公司 亚氨基糖以及治疗病毒性疾病的方法
KR101787434B1 (ko) * 2009-02-24 2017-11-15 유나이티드 세러퓨틱스 코오포레이션 이미노슈가 및 아레나바이러스성 감염을 치료하는 방법
WO2010144759A1 (en) 2009-06-12 2010-12-16 United Therapeutics Corporation Iminosugars and methods of treating bunyaviral and togaviral diseases
KR101459530B1 (ko) * 2009-09-04 2014-11-07 유나이티드 세러퓨틱스 코오포레이션 폭스바이러스 감염의 치료 방법
PL2473482T3 (pl) * 2009-09-04 2014-09-30 United Therapeutics Corp Metody leczenia zakażeń ortomyksowirusowych
EP2473046B1 (en) * 2009-09-04 2014-10-22 United Therapeutics Corporation Iminosugars for their use in the treatment of filoviral diseases
EP3552664A1 (en) 2011-05-12 2019-10-16 Proteostasis Therapeutics, Inc. Proteostasis regulators
JP6180423B2 (ja) * 2011-11-08 2017-08-16 ユニヴァーシティ オブ ワシントン リソソーム酵素アッセイ法および組成物
EP2806875B1 (en) 2012-01-25 2017-07-19 Proteostasis Therapeutics, Inc. Proteasome activity modulating compounds
KR20210062731A (ko) * 2012-03-07 2021-05-31 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
ES2859761T3 (es) 2012-05-03 2021-10-04 Amicus Therapeutics Inc Pautas posológicas para el tratamiento de enfermedad de Pompe
WO2014017915A2 (en) 2012-07-27 2014-01-30 Universiteit Utrecht Holding B.V. Urea and guanidinium derivatives of iminosugars
CN102875450B (zh) * 2012-10-25 2015-04-08 上海丝绸集团股份有限公司 一种从桑叶中提取制备1-脱氧野尻霉素的工艺方法
MX2015008837A (es) * 2013-01-09 2015-10-14 Amicus Therapeutics Inc Composiciones parenterales estables de dnj.
PL3201320T3 (pl) 2014-09-30 2024-03-18 Amicus Therapeutics, Inc. Bardzo silnie działająca kwasowa alfa-glukozydaza ze zwiększoną zawartością węglowodanów
CN107207477B (zh) 2014-11-05 2022-11-01 伊美根特病毒学公司 用于治疗病毒性疾病的亚氨基糖
PT3397273T (pt) 2015-12-30 2021-08-09 Amicus Therapeutics Inc Alfa-glicosidase ácida aumentada para o tratamento da doença de pompe
WO2017117407A1 (en) 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
TWI823272B (zh) * 2016-03-30 2023-11-21 美商阿米庫斯醫療股份有限公司 用於選擇高m6p重組蛋白之方法
SG11201808455VA (en) 2016-03-30 2018-10-30 Amicus Therapeutics Inc Formulations comprising recombinant acid alpha-glucosidase
PE20190127A1 (es) 2016-03-30 2019-01-17 Amicus Therapeutics Inc Metodo para seleccionar proteinas recombinantes ricas en m6p
BR112018071684A2 (pt) 2016-04-21 2019-02-19 Baylor College Of Medicine composições e métodos para o tratamento de doenças ou distúrbios de armazenamento lisossômico que tem como característica disfunção lisossomal
JP2017195786A (ja) * 2016-04-25 2017-11-02 国立大学法人 熊本大学 細胞内酵素タンパク質の構造異常の正常化方法、細胞内酵素タンパク質の構造異常の検出方法、及び、遺伝性代謝病の治療方法ならびにその治療効果の予測・評価方法
AU2017260705A1 (en) * 2016-05-04 2018-11-22 Mirexus Biotechnologies Inc. Glycogen and phytoglycogen nanoparticles as immunosuppressive compounds, and compositions and methods of use thereof
EA202290114A1 (ru) * 2017-03-30 2022-03-29 Амикус Терапьютикс, Инк. Способ отбора рекомбинантных белков с высоким содержанием m6p
MX2019014427A (es) 2017-06-01 2020-02-05 Idorsia Pharmaceuticals Ltd Forma cristalina de n-butildeoxigalactonojirimicina.
BR112020026580A2 (pt) 2018-06-27 2021-03-23 Proteostasis Therapeutics, Inc. (Fem) Compostos, composição farmacêutica e métodos para tratar um paciente
JP2021530465A (ja) 2018-06-27 2021-11-11 プロテオステイシス セラピューティクス， インコーポレイテッド プロテアソーム活性増強化合物
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
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
JP2022540632A (ja) 2019-07-09 2022-09-16 ジェネトン 糖原病（ｇｓｄ）の処置
TWI759888B (zh) * 2019-10-04 2022-04-01 中央研究院 用以治療龐貝氏症之方法
EP3871687A1 (en) 2020-02-27 2021-09-01 eleva GmbH Enzyme replacement therapy for treating pompe disease
CR20220601A (es) * 2020-05-07 2023-04-11 Alectos Therapeutics Inc Inhibidores de glucosilceramidasa no lisosomal y los usos de los mismos
EP3944858A1 (en) 2020-07-30 2022-02-02 Beyond Batten Disease Foundation Combination product containing trehalose and miglustat for treating lysosomal diseases
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
WO2022227287A1 (zh) * 2021-04-25 2022-11-03 中南大学 Dnj及其衍生物在制备预防和/或治疗肺动脉高压药物中的应用

Family Cites Families (89)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US430307A (en) 1890-06-17 Reversible plow
NO154918C (no) 1977-08-27 1987-01-14 Bayer Ag Analogifremgangsmaate til fremstilling av terapeutisk aktive derivater av 3,4,5-trihydroksypiperidin.
DE2834122A1 (de) 1978-08-03 1980-02-14 Bayer Ag Verfahren zur herstellung von 6-amino-6-desoxy-l-sorbose
DE2853573A1 (de) 1978-12-12 1980-07-03 Bayer Ag Herstellung von n-substituierten derivaten des l-desoxynojirimycins
DE3038901A1 (de) 1980-10-15 1982-05-06 Bayer Ag, 5090 Leverkusen Verfahren zur herstellung von n-substituierten derivaten des 1-desoxynojirimycins
US4407957A (en) 1981-03-13 1983-10-04 Damon Corporation Reversible microencapsulation of a core material
US4980286A (en) 1985-07-05 1990-12-25 Whitehead Institute For Biomedical Research In vivo introduction and expression of foreign genetic material in epithelial cells
US4837237A (en) * 1985-07-09 1989-06-06 Fred Hutchinson Cancer Research Center Therapy using glucosidase processing inhibitors
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
DE3611841A1 (de) 1986-04-09 1987-10-15 Bayer Ag Verfahren zur herstellung von 1-desoxynojirimycin und dessen n-derivaten
DE3737523A1 (de) 1987-11-05 1989-05-18 Bayer Ag Verwendung von substituierten hydroxypiperidinen als antivirale mittel
US5879680A (en) 1987-12-23 1999-03-09 The United States Of America As Represented By The Department Of Health And Human Services Cloned DNA for synthesizing unique glucocerebrosidase
US5672344A (en) 1987-12-30 1997-09-30 The Regents Of The University Of Michigan Viral-mediated gene transfer system
US4985445A (en) * 1988-02-12 1991-01-15 Meiji Seika Kaisha, Ltd. Cancer cell metastasis inhibitors and novel compounds
US4880917A (en) 1988-07-01 1989-11-14 Merrell Dow Pharmaceuticals Inc. Process for hydrolyzing 2,6-dideoxy-2,6-iminoheptononitrile derivatives using trifluoroacetic acid and dinitrogen tetroxide
US5310745A (en) 1988-11-03 1994-05-10 G. D. Searle & Co. Antiviral compounds
US5236838A (en) 1988-12-23 1993-08-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US6451600B1 (en) 1989-12-22 2002-09-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5179023A (en) 1989-03-24 1993-01-12 Research Corporation Technologies, Inc. Recombinant α-galactosidase a therapy for Fabry disease
US5011829A (en) * 1989-06-02 1991-04-30 G. D. Searle & Co. Pharmaceutical composition and method of inhibiting virus
US5103008A (en) * 1989-08-17 1992-04-07 Monsanto Company Compound, N-butyl-deoxynojirimycin-6-phosphate
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US5670488A (en) 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
CA2092323A1 (en) 1990-10-01 1992-04-02 George Y. Wu Targeting viruses and cells for selective internalization by cells
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
AU666118B2 (en) 1991-04-25 1996-02-01 Brown University Research Foundation Implantable biocompatible immunoisolatory vehicle for delivery of selected therapeutic products
AU668870B2 (en) 1991-05-14 1996-05-23 Targetech, Inc Targeted delivery of genes encoding immunogenic proteins
DK0587738T3 (da) 1991-06-05 2000-12-18 Univ Connecticut Destineret levering af gener, som koder for sekretionsproteiner
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
PT101031B (pt) 1991-11-05 2002-07-31 Transkaryotic Therapies Inc Processo para o fornecimento de proteinas por terapia genetica
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
WO1993014188A1 (en) 1992-01-17 1993-07-22 The Regents Of The University Of Michigan Targeted virus
US5401645A (en) 1992-03-16 1995-03-28 Monsanto Company Process for producing n-substituted polyhydroxy nitrogen-containing heterocycles utilizing acetobacteraceae and corynebacterium
US5383851A (en) 1992-07-24 1995-01-24 Bioject Inc. Needleless hypodermic injection device
US5569189A (en) 1992-09-28 1996-10-29 Equidyne Systems, Inc. hypodermic jet injector
JP3717925B2 (ja) 1992-09-29 2005-11-16 ネクター セラピューティクス 副甲状腺ホルモンの活性フラグメントの肺導入
US6291657B1 (en) 1993-05-13 2001-09-18 Monsanto Company Deoxygalactonojirimycin derivatives
US5399567A (en) * 1993-05-13 1995-03-21 Monsanto Company Method of treating cholera
US5798366A (en) 1993-05-13 1998-08-25 Monsanto Company Method for treatment of CNS-involved lysosomal storage diseases
US5635493A (en) 1993-12-01 1997-06-03 Marine Polymer Technologies, Inc. Methods and compositions for poly-β-1-4-N-acetylglucosamine chemotherapeutics
WO1995022975A1 (en) 1994-02-25 1995-08-31 G.D. Searle & Co. Use of 1-deoxynojirimycin and its derivatives for treating mammals infected with respiratory syncytial virus
FR2718357B1 (fr) 1994-04-06 1997-10-03 Defarges Alain Moreau Perfectionnements apportés à un dispositif d'injection par jet sans aiguille.
US5767099A (en) 1994-12-09 1998-06-16 Genzyme Corporation Cationic amphiphiles containing amino acid or dervatized amino acid groups for intracellular delivery of therapeutic molecules
US5939401A (en) 1994-12-09 1999-08-17 Genzyme Corporation Cationic amphiphile compositions for intracellular delivery of therapeutic molecules
US6331524B1 (en) 1994-12-09 2001-12-18 Genzyme Corporation Organ-specific targeting of cationic amphiphile / DNA complexes for gene therapy
US5599302A (en) 1995-01-09 1997-02-04 Medi-Ject Corporation Medical injection system and method, gas spring thereof and launching device using gas spring
US5780014A (en) 1995-04-14 1998-07-14 Inhale Therapeutic Systems Method and apparatus for pulmonary administration of dry powder alpha 1-antitrypsin
US5654007A (en) 1995-06-07 1997-08-05 Inhale Therapeutic Systems Methods and system for processing dispersible fine powders
US20030007963A1 (en) 1998-12-07 2003-01-09 Van Bree Johannes B. M. M. Treatment of pompe's disease
US6118045A (en) 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
CA2230758A1 (en) 1995-09-08 1997-03-13 Genzyme Corporation Improved aav vectors for gene therapy
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
US6270954B1 (en) 1996-04-10 2001-08-07 The Regents Of The University Of California Correction of genetic defects using chemical chaperones
US6458574B1 (en) 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
US6083725A (en) 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
US5948653A (en) 1997-03-21 1999-09-07 Pati; Sushma Sequence alterations using homologous recombination
US6210666B1 (en) 1997-10-21 2001-04-03 Orphan Medical, Inc. Truncated α-galactosidase A to treat fabry disease
AU753336B2 (en) * 1997-11-10 2002-10-17 G.D. Searle & Co. Use of alkylated iminosugars to treat multidrug resistance
US6465488B1 (en) 1997-12-11 2002-10-15 Chancellor, Masters & Scholars Of The University Of Oxford Inhibition of glycolipid biosynthesis
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
WO2000047198A2 (en) * 1999-02-12 2000-08-17 G.D. Searle & Co. Use of substituted-1,5-dideoxy-1,5-imino-d-glucitol compounds for treating hepatitis virus infections
AUPP942599A0 (en) 1999-03-24 1999-04-15 Commonwealth Scientific And Industrial Research Organisation Antiviral agents
WO2001007078A1 (en) * 1999-07-26 2001-02-01 G.D. Searle & Co. Use of long-chain n-alkyl derivates of deoxynojirimycin and a glucocerebrosidase enzyme for the manufacture of medicament for the treatment of glycolipid storage diseases
US6537785B1 (en) 1999-09-14 2003-03-25 Genzyme Glycobiology Research Institute, Inc. Methods of treating lysosomal storage diseases
US6244113B1 (en) 1999-10-29 2001-06-12 University Of Alabama In Huntsville Method and apparatus for measuring microgravity acceleration
US6188045B1 (en) 2000-04-03 2001-02-13 Alto-Shaam, Inc. Combination oven with three-stage water atomizer
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20020095135A1 (en) * 2000-06-19 2002-07-18 David Meeker Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
CN1638739A (zh) * 2000-08-18 2005-07-13 法玛西雅厄普约翰美国公司 治疗成瘾性障碍的化合物
ES2300439T3 (es) 2001-04-30 2008-06-16 Zystor Therapeutics , Inc. Reconocimiento subcelular de proteinas terapeuticas.
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
WO2003086452A2 (en) 2002-04-05 2003-10-23 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy
WO2004037373A2 (en) 2002-10-21 2004-05-06 The Scripps Research Institute CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF β-GLUCOSIDASE
SI2857036T1 (sl) 2003-01-31 2017-05-31 Mount Sinai School Of Medicine Of New York University Kombinirana terapija za zdravljenje motenj pomanjkanja proteinov
FR2861991B1 (fr) 2003-11-07 2008-01-18 Centre Nat Rech Scient Utilisation d'inhibiteurs de glucosidase pour une therapie de la mucoviscidose
WO2005063706A1 (en) 2003-12-23 2005-07-14 Macrozyme Dnm B.V. Synthesis of nojirimycins
JP2007523648A (ja) 2004-02-06 2007-08-23 バイオマリン ファーマシューティカル インコーポレイテッド 高リン酸化リソソーム酵素製剤及びそれらの使用
CA2553955C (en) 2004-02-10 2012-08-28 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
EP1896083B1 (en) 2005-05-17 2016-03-02 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
EP2099523A2 (en) 2006-11-13 2009-09-16 ZyStor Therapeutics , Inc. Methods for treating pompe disease
WO2008112525A2 (en) 2007-03-09 2008-09-18 Link Medicine Corporation Treatment of lysosomal storage diseases
US20100317690A1 (en) 2007-11-21 2010-12-16 Summit Corporation Plc Treatment of protein folding disorders
CA2718059A1 (en) 2008-03-12 2009-09-17 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for pompe disease
WO2010015816A2 (en) 2008-08-06 2010-02-11 Summit Corporation Plc Treatment of lysosomal storage disorders and other proteostatic diseases
US20100119502A1 (en) 2008-11-11 2010-05-13 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
EP2416656B1 (en) 2009-04-09 2015-12-23 Amicus Therapeutics, Inc. Methods for preventing and/or treating lysosomal storage disorders
EP2475376B1 (en) 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
CN104409036B (zh) 2014-12-16 2017-01-11 上海天马微电子有限公司 一种显示面板和显示装置

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