JP2023512366A - リソソーム蓄積性疾患と関連する神経学的症状を処置するための方法 - Google Patents

リソソーム蓄積性疾患と関連する神経学的症状を処置するための方法 Download PDF

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JP2023512366A
JP2023512366A JP2021564953A JP2021564953A JP2023512366A JP 2023512366 A JP2023512366 A JP 2023512366A JP 2021564953 A JP2021564953 A JP 2021564953A JP 2021564953 A JP2021564953 A JP 2021564953A JP 2023512366 A JP2023512366 A JP 2023512366A
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brain
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brain tissue
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ナイジェル・パトリック・ソマーヴィル・クロフォード
ターニャ・ザレンバ・フィッシャー
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Genzyme Corp
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    • A—HUMAN NECESSITIES
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    • A61B5/00—Measuring for diagnostic purposes; Identification of persons
    • A61B5/05—Detecting, measuring or recording for diagnosis by means of electric currents or magnetic fields; Measuring using microwaves or radio waves
    • A61B5/055—Detecting, measuring or recording for diagnosis by means of electric currents or magnetic fields; Measuring using microwaves or radio waves involving electronic [EMR] or nuclear [NMR] magnetic resonance, e.g. magnetic resonance imaging
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61B—DIAGNOSIS; SURGERY; IDENTIFICATION
    • A61B6/00—Apparatus or devices for radiation diagnosis; Apparatus or devices for radiation diagnosis combined with radiation therapy equipment
    • A61B6/02—Arrangements for diagnosis sequentially in different planes; Stereoscopic radiation diagnosis
    • A61B6/03—Computed tomography [CT]
    • A61B6/037—Emission tomography
    • 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
    • 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
    • A61K31/47—Quinolines; Isoquinolines
    • A61K31/4748—Quinolines; Isoquinolines forming part of bridged ring systems
    • 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
    • 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
    • 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
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • 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

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Wood Science & Technology (AREA)
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JP2021564953A 2020-02-03 2021-02-03 リソソーム蓄積性疾患と関連する神経学的症状を処置するための方法 Pending JP2023512366A (ja)

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JP2025153760A JP2026001009A (ja) 2020-02-03 2025-09-17 リソソーム蓄積性疾患と関連する神経学的症状を処置するための方法

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US202062969568P 2020-02-03 2020-02-03
US62/969,568 2020-02-03
US202063029154P 2020-05-22 2020-05-22
US63/029,154 2020-05-22
US202063072714P 2020-08-31 2020-08-31
US63/072,714 2020-08-31
PCT/IB2021/050879 WO2021156769A1 (en) 2020-02-03 2021-02-03 Methods for treating neurological symptoms associated with lysosomal storage diseases

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JP2023512366A5 JP2023512366A5 (https=) 2024-02-13

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JP2025153760A Pending JP2026001009A (ja) 2020-02-03 2025-09-17 リソソーム蓄積性疾患と関連する神経学的症状を処置するための方法

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US (1) US12083115B2 (https=)
EP (1) EP4100009A1 (https=)
JP (2) JP2023512366A (https=)
KR (1) KR20220136887A (https=)
CN (1) CN115427038A (https=)
AU (1) AU2021215396B2 (https=)
BR (1) BR112022014553A2 (https=)
CA (1) CA3166777A1 (https=)
IL (1) IL295255A (https=)
MX (1) MX2022009477A (https=)
TW (1) TW202142236A (https=)
WO (1) WO2021156769A1 (https=)

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MA37975B2 (fr) 2012-09-11 2021-03-31 Genzyme Corp Inhibiteurs de synthase de glucosylcéramide
CN114040762A (zh) 2019-02-04 2022-02-11 建新公司 使用葡糖神经酰胺合酶(gcs)的抑制剂治疗纤毛疾病
JP2023512366A (ja) 2020-02-03 2023-03-27 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神経学的症状を処置するための方法
AU2021311131B2 (en) 2020-07-24 2026-02-19 Genzyme Corporation Pharmaceutical compositions comprising venglustat
WO2025219952A1 (en) * 2024-04-19 2025-10-23 Genzyme Corporation Venglustat for use in methods for reducing disease related biomarker levels in patients with ganglioside storage disorders
WO2025219951A1 (en) * 2024-04-19 2025-10-23 Genzyme Corporation Venglustat for use in methods for reducing disease related biomarker levels in patients with ganglioside storage disorders

Citations (1)

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JP2014517808A (ja) * 2011-03-18 2014-07-24 ジェンザイム・コーポレーション グルコシルセラミド合成酵素阻害剤

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