JP6990110B2 - タンパク質症を処置するための方法 - Google Patents

タンパク質症を処置するための方法 Download PDF

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Publication number
JP6990110B2
JP6990110B2 JP2017547448A JP2017547448A JP6990110B2 JP 6990110 B2 JP6990110 B2 JP 6990110B2 JP 2017547448 A JP2017547448 A JP 2017547448A JP 2017547448 A JP2017547448 A JP 2017547448A JP 6990110 B2 JP6990110 B2 JP 6990110B2
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compound
quinuclidine
mice
disease
proteinosis
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JP2017547448A
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Japanese (ja)
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JP2018507886A (ja
JP2018507886A5 (enExample
Inventor
エイチ・セン・チェン
ラムヤ・シハブディン
パブロ・セルジオ・サルディ
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Genzyme Corp
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Genzyme Corp
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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
    • A61K31/439—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 the ring forming part of a bridged ring system, e.g. quinuclidine
    • 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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D453/00—Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids
    • C07D453/02—Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids containing not further condensed quinuclidine ring systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
JP2017547448A 2015-03-10 2016-03-09 タンパク質症を処置するための方法 Active JP6990110B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2021078459A JP7374149B2 (ja) 2015-03-10 2021-05-06 タンパク質症を処置するための方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562131071P 2015-03-10 2015-03-10
US62/131,071 2015-03-10
PCT/US2016/021512 WO2016145046A1 (en) 2015-03-10 2016-03-09 Methods for treating proteinopathies

Related Child Applications (1)

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JP2021078459A Division JP7374149B2 (ja) 2015-03-10 2021-05-06 タンパク質症を処置するための方法

Publications (3)

Publication Number Publication Date
JP2018507886A JP2018507886A (ja) 2018-03-22
JP2018507886A5 JP2018507886A5 (enExample) 2019-04-25
JP6990110B2 true JP6990110B2 (ja) 2022-02-10

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JP2017547448A Active JP6990110B2 (ja) 2015-03-10 2016-03-09 タンパク質症を処置するための方法
JP2021078459A Active JP7374149B2 (ja) 2015-03-10 2021-05-06 タンパク質症を処置するための方法

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JP2021078459A Active JP7374149B2 (ja) 2015-03-10 2021-05-06 タンパク質症を処置するための方法

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US (2) US20180036295A1 (enExample)
EP (2) EP3267983B1 (enExample)
JP (2) JP6990110B2 (enExample)
KR (1) KR20170123329A (enExample)
CN (1) CN107872976A (enExample)
AU (1) AU2016229826A1 (enExample)
CA (1) CA2978883A1 (enExample)
EA (1) EA201791993A1 (enExample)
ES (1) ES2973101T3 (enExample)
HK (1) HK1244217A1 (enExample)
HU (1) HUE065628T2 (enExample)
IL (1) IL254393A0 (enExample)
MX (1) MX2017011598A (enExample)
PL (1) PL3267983T3 (enExample)
PT (1) PT3267983T (enExample)
TW (1) TW201642855A (enExample)
WO (1) WO2016145046A1 (enExample)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JOP20130273B1 (ar) 2012-09-11 2021-08-17 Genzyme Corp مثبطات انزيم (سينثاز) غلوكوسيل سيراميد
BR112019027029A2 (pt) * 2017-06-19 2020-07-14 Kainos Medicine, Inc. moduladores de alfa-sinucleina
TWI644673B (zh) * 2017-07-27 2018-12-21 百朗克股份有限公司 頭孢曲松的用途
JP7633169B2 (ja) * 2019-02-04 2025-02-19 ジェンザイム・コーポレーション グルコシルセラミドシンターゼ（ｇｃｓ）阻害剤を使用した繊毛病の処置
JP7511567B2 (ja) * 2019-02-04 2024-07-05 ジェンザイム・コーポレーション リソソーム蓄積性疾患と関連する症状および障害を処置するための方法
ES2984255T3 (es) * 2019-11-15 2024-10-29 Yuhan Corp Derivados que tienen un resto 2,3-dihidro-1H-indeno o 2,3-dihidrobenzofurano o una sal farmacéuticamente aceptable de mismo y composiciones farmacéuticas que comprenden los mismos
US12083115B2 (en) * 2020-02-03 2024-09-10 Genzyme Corporation Methods for treating neurological symptoms associated with lysosomal storage diseases
JP7637121B2 (ja) * 2020-03-17 2025-02-27 住友ファーマ株式会社 オキサジアゾール誘導体
CN115715189A (zh) * 2020-03-23 2023-02-24 普拉西斯精密医药公司 Kcnt1抑制剂和使用方法
BR112023000798A2 (pt) * 2020-07-24 2023-02-07 Genzyme Corp Composições farmacêuticas compreendendo venglustat
CA3187086A1 (en) * 2020-07-30 2022-02-03 Tanya Zaremba FISCHER Methods for reducing glycosphingolipid concentration in brain tissue and methods of treatment of neurodegenerative diseases involving the same
US11773088B2 (en) 2020-11-02 2023-10-03 Praxis Precision Medicines, Inc. KCNT1 inhibitors and methods of use
AU2023403087A1 (en) 2022-12-01 2025-07-17 Genzyme Corporation Venglustat in combination with a strong or moderate inhibitor of cyp3a4
WO2025262570A1 (en) 2024-06-17 2025-12-26 Genzyme Corporation Venglustat as an inhibitor of glucosylceramide synthase in subjects having hepatic impairment

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014043068A1 (en) 2012-09-11 2014-03-20 Genzyme Corporation Glucosylceramide synthase inhibitors

Family Cites Families (10)

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Publication number Priority date Publication date Assignee Title
US4683A (en) 1846-08-08 waring and richard e
US195A (en) 1837-05-15 Machine fob cutting and dressing stone
BE640616A (enExample) 1962-12-19
US3492397A (en) 1967-04-07 1970-01-27 Warner Lambert Pharmaceutical Sustained release dosage in the pellet form and process thereof
US4060598A (en) 1967-06-28 1977-11-29 Boehringer Mannheim G.M.B.H. Tablets coated with aqueous resin dispersions
US3538214A (en) 1969-04-22 1970-11-03 Merck & Co Inc Controlled release medicinal tablets
US4173626A (en) 1978-12-11 1979-11-06 Merck & Co., Inc. Sustained release indomethacin
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
PT2685986T (pt) * 2011-03-18 2020-03-05 Genzyme Corp Inibidor de glicosilceramida-sintase
CA2840224C (en) * 2011-06-22 2019-08-13 The General Hospital Corporation Treatment of proteinopathies

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014043068A1 (en) 2012-09-11 2014-03-20 Genzyme Corporation Glucosylceramide synthase inhibitors

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
J Neurochem，2014年，129(5)，884-894

Also Published As

Publication number Publication date
IL254393A0 (en) 2017-11-30
JP2021119185A (ja) 2021-08-12
WO2016145046A1 (en) 2016-09-15
JP2018507886A (ja) 2018-03-22
PL3267983T3 (pl) 2024-05-13
KR20170123329A (ko) 2017-11-07
JP7374149B2 (ja) 2023-11-06
CA2978883A1 (en) 2016-09-15
TW201642855A (zh) 2016-12-16
US20180036295A1 (en) 2018-02-08
EP4349408A2 (en) 2024-04-10
PT3267983T (pt) 2024-03-12
HUE065628T2 (hu) 2024-06-28
AU2016229826A1 (en) 2017-10-26
ES2973101T3 (es) 2024-06-18
MX2017011598A (es) 2017-12-20
US20200197374A1 (en) 2020-06-25
EA201791993A1 (ru) 2017-12-29
CN107872976A (zh) 2018-04-03
EP3267983A1 (en) 2018-01-17
HK1244217A1 (zh) 2018-08-03
EP4349408A3 (en) 2024-06-19
EP3267983B1 (en) 2023-12-20

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