HK1243643A1 - 用於治疗和/或预防脑淀粉样变性的给药方案 - Google Patents

用於治疗和/或预防脑淀粉样变性的给药方案 Download PDF

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Publication number
HK1243643A1
HK1243643A1 HK18103256.3A HK18103256A HK1243643A1 HK 1243643 A1 HK1243643 A1 HK 1243643A1 HK 18103256 A HK18103256 A HK 18103256A HK 1243643 A1 HK1243643 A1 HK 1243643A1
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HK
Hong Kong
Prior art keywords
addnj
period
enzyme
administration
formulation
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HK18103256.3A
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English (en)
Chinese (zh)
Inventor
布拉登.艾伦.乌斯特曼
布拉登.艾倫.烏斯特曼
Original Assignee
阿米库斯治疗学公司
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Publication of HK1243643A1 publication Critical patent/HK1243643A1/zh

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    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
HK18103256.3A 2014-04-14 2015-04-14 用於治疗和/或预防脑淀粉样变性的给药方案 HK1243643A1 (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461979408P 2014-04-14 2014-04-14
US61/979,408 2014-04-14
US14/684,967 2015-04-13
US14/684,967 US9675627B2 (en) 2014-04-14 2015-04-13 Dosing regimens for treating and/or preventing cerebral amyloidoses
PCT/US2015/025733 WO2015160796A1 (en) 2014-04-14 2015-04-14 Dosing regimens for treating and/or preventing cerebral amyloidoses

Publications (1)

Publication Number Publication Date
HK1243643A1 true HK1243643A1 (zh) 2018-07-20

Family

ID=53015935

Family Applications (1)

Application Number Title Priority Date Filing Date
HK18103256.3A HK1243643A1 (zh) 2014-04-14 2015-04-14 用於治疗和/或预防脑淀粉样变性的给药方案

Country Status (10)

Country Link
US (2) US9675627B2 (enExample)
EP (1) EP3131547A1 (enExample)
JP (1) JP6662789B2 (enExample)
KR (1) KR20170003576A (enExample)
CN (1) CN107148270A (enExample)
AU (1) AU2015247859B2 (enExample)
CA (1) CA2945707A1 (enExample)
HK (1) HK1243643A1 (enExample)
SG (1) SG11201610349SA (enExample)
WO (1) WO2015160796A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6061677B2 (ja) * 2009-10-19 2017-01-18 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てガングリオシドの活性を増大させて、アルツハイマー病を治療するための方法
US9994589B2 (en) 2015-12-14 2018-06-12 Amicus Therapeutics, Inc. Compounds and methods for the treatment of alzheimer's disease and/or cerebral amyloid angiopathy
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

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4407957A (en) 1981-03-13 1983-10-04 Damon Corporation Reversible microencapsulation of a core material
SG47470A1 (en) 1991-04-25 1998-04-17 Univ Brown Res Found Implantable biocompatible immunoisolatory vehicle for delivery of a selected therapeutic products
US5766846A (en) 1992-07-10 1998-06-16 Athena Neurosciences Methods of screening for compounds which inhibit soluble β-amyloid peptide production
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
ATE220327T1 (de) 1992-09-29 2002-07-15 Inhale Therapeutic Syst Pulmonale abgabe von aktiven fragmenten des parathormons
FR2718357B1 (fr) 1994-04-06 1997-10-03 Defarges Alain Moreau Perfectionnements apportés à un dispositif d'injection par jet sans aiguille.
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
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
US20050031651A1 (en) 2002-12-24 2005-02-10 Francine Gervais Therapeutic formulations for the treatment of beta-amyloid related diseases
WO2004103368A1 (en) 2003-05-22 2004-12-02 The Hospital For Sick Children Treatment of tay sachs or sandhoff diseases by enhancing hexosaminidase activity
MX2007015602A (es) 2005-06-08 2008-02-21 Amicus Therapeutics Inc Tratamiento de trastornos del sistema nervioso central asociados con mutaciones en genes que codifican las enzimas lisosomicas.
JP2009537149A (ja) * 2006-05-16 2009-10-29 アミークス セラピューティックス インコーポレイテッド ファブリー病に対する治療の選択を診断し、評価するためのアッセイ
ATE555788T1 (de) 2006-06-23 2012-05-15 Amicus Therapeutics Inc Verfahren zur behandlung neurologischer erkrankungen mittels verstärkung der beta - glucocerebrosidaseaktivität
WO2008134628A2 (en) 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
WO2011011181A1 (en) 2009-07-01 2011-01-27 Amicus Therapeutics, Inc. Pharmacological chaperones for the treatment of alzheimer's disease
JP6061677B2 (ja) 2009-10-19 2017-01-18 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てガングリオシドの活性を増大させて、アルツハイマー病を治療するための方法
US20140219986A1 (en) * 2011-03-11 2014-08-07 Amicus Therapeutics ,Inc. Dosing regimens for the treatment of fabry disease
ES2864774T3 (es) * 2011-12-22 2021-10-14 Centogene Ip Gmbh Combinación de un compuesto que tiene la capacidad de reorganizar una enzima lisosomal y Ambroxol y/o un derivado de Ambroxol
EP2844279B1 (en) * 2012-05-03 2020-12-09 Amicus Therapeutics, Inc. Dosing regimens for the treatment of pompe disease

Also Published As

Publication number Publication date
AU2015247859A1 (en) 2016-12-01
CN107148270A (zh) 2017-09-08
US9675627B2 (en) 2017-06-13
JP6662789B2 (ja) 2020-03-11
AU2015247859B2 (en) 2020-03-26
JP2017511372A (ja) 2017-04-20
KR20170003576A (ko) 2017-01-09
WO2015160796A1 (en) 2015-10-22
US20160008383A1 (en) 2016-01-14
CA2945707A1 (en) 2015-10-22
US20170273996A1 (en) 2017-09-28
EP3131547A1 (en) 2017-02-22
SG11201610349SA (en) 2017-01-27

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