CN107148270A - 用于治疗和/或预防脑淀粉样变性的给药方案 - Google Patents

用于治疗和/或预防脑淀粉样变性的给药方案 Download PDF

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Publication number
CN107148270A
CN107148270A CN201580031471.1A CN201580031471A CN107148270A CN 107148270 A CN107148270 A CN 107148270A CN 201580031471 A CN201580031471 A CN 201580031471A CN 107148270 A CN107148270 A CN 107148270A
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CN
China
Prior art keywords
addnj
period
enzyme
boost
administered
Prior art date
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Pending
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CN201580031471.1A
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English (en)
Chinese (zh)
Inventor
布拉登·艾伦·乌斯特曼
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Amicus Therapeutics Inc
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Amicus Therapeutics Inc
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Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of CN107148270A publication Critical patent/CN107148270A/zh
Pending legal-status Critical Current

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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
    • A61K31/70—Carbohydrates; Sugars; 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs 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)
CN201580031471.1A 2014-04-14 2015-04-14 用于治疗和/或预防脑淀粉样变性的给药方案 Pending CN107148270A (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 US9675627B2 (en) 2014-04-14 2015-04-13 Dosing regimens for treating and/or preventing cerebral amyloidoses
US14/684,967 2015-04-13
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
CN107148270A true CN107148270A (zh) 2017-09-08

Family

ID=53015935

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201580031471.1A Pending CN107148270A (zh) 2014-04-14 2015-04-14 用于治疗和/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
PL2490712T3 (pl) * 2009-10-19 2015-12-31 Amicus Therapeutics Inc Sposób leczenia choroby Alzheimera przy zastosowaniu chaperonów farmakologicznych dla zwiększenia aktywności gangliozydaz
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
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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011049787A1 (en) * 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases

Family Cites Families (21)

* 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
EP0585368B1 (en) 1991-04-25 1997-08-06 Brown University Research Foundation Implantable biocompatible immunoisolatory vehicle for delivery of 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
DK0679088T3 (da) 1992-09-29 2002-10-28 Inhale Therapeutic Syst Pulmonal levering af aktive fragmenter af parathyreoideaea hormon
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
US20070066543A1 (en) 2003-05-22 2007-03-22 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.
EP2024745B1 (en) * 2006-05-16 2015-07-08 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
JP5303458B2 (ja) 2006-06-23 2013-10-02 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 β−グルコセレブロシダーゼの活性増強による神経学的疾患の治療方法
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.
WO2011011181A1 (en) 2009-07-01 2011-01-27 Amicus Therapeutics, Inc. Pharmacological chaperones for the treatment of alzheimer's disease
EA031874B1 (ru) * 2011-03-11 2019-03-29 Амикус Терапьютикс, Инк. Способ лечения болезни фабри
AU2012359165A1 (en) * 2011-12-22 2014-07-10 Centogene Ip Gmbh Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol
HUE053565T2 (hu) * 2012-05-03 2021-07-28 Amicus Therapeutics Inc Adagolási rendek Pompe betegség kezelésére

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011049787A1 (en) * 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases

Also Published As

Publication number Publication date
EP3131547A1 (en) 2017-02-22
SG11201610349SA (en) 2017-01-27
JP2017511372A (ja) 2017-04-20
AU2015247859B2 (en) 2020-03-26
WO2015160796A1 (en) 2015-10-22
US9675627B2 (en) 2017-06-13
US20160008383A1 (en) 2016-01-14
HK1243643A1 (zh) 2018-07-20
CA2945707A1 (en) 2015-10-22
JP6662789B2 (ja) 2020-03-11
US20170273996A1 (en) 2017-09-28
KR20170003576A (ko) 2017-01-09
AU2015247859A1 (en) 2016-12-01

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