CN114040762A - 使用葡糖神经酰胺合酶(gcs)的抑制剂治疗纤毛疾病 - Google Patents

使用葡糖神经酰胺合酶(gcs)的抑制剂治疗纤毛疾病 Download PDF

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Publication number
CN114040762A
CN114040762A CN202080025851.5A CN202080025851A CN114040762A CN 114040762 A CN114040762 A CN 114040762A CN 202080025851 A CN202080025851 A CN 202080025851A CN 114040762 A CN114040762 A CN 114040762A
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compound
disease
syndrome
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O·易卜拉欣莫夫·贝斯克罗夫纳亚
N·O·布卡诺夫
H·休松
S·E·莫雷诺
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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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

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  • Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202080025851.5A 2019-02-04 2020-02-04 使用葡糖神经酰胺合酶(gcs)的抑制剂治疗纤毛疾病 Pending CN114040762A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962800993P 2019-02-04 2019-02-04
US62/800,993 2019-02-04
US201962851430P 2019-05-22 2019-05-22
US62/851,430 2019-05-22
PCT/US2020/016588 WO2020163337A1 (en) 2019-02-04 2020-02-04 Treatment of ciliopathies using inhibitors of glucosylceramide synthase (gcs)

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CN114040762A true CN114040762A (zh) 2022-02-11

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CN202080025851.5A Pending CN114040762A (zh) 2019-02-04 2020-02-04 使用葡糖神经酰胺合酶(gcs)的抑制剂治疗纤毛疾病

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US (1) US12527776B2 (https=)
EP (1) EP3920912B1 (https=)
JP (1) JP7633169B2 (https=)
CN (1) CN114040762A (https=)
AU (1) AU2020218185B2 (https=)
ES (1) ES3040555T3 (https=)
HU (1) HUE072626T2 (https=)
PL (1) PL3920912T3 (https=)
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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
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EP4201403A1 (en) 2021-12-21 2023-06-28 Som Innovation Biotech, S.L. Compounds tirapazamine and quazinone for use in the treatment of gm2 gangliosidoses
JP2025541739A (ja) 2022-12-01 2025-12-23 ジェンザイム・コーポレーション Ｃｙｐ３ａ４の強力な又は中程度の阻害剤と組み合わせたベングルスタット
WO2025178019A1 (ja) * 2024-02-19 2025-08-28 国立大学法人大阪大学 繊毛病治療用医薬組成物
WO2025262570A1 (en) 2024-06-17 2025-12-26 Genzyme Corporation Venglustat as an inhibitor of glucosylceramide synthase in subjects having hepatic impairment

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WO2020163337A1 (en) 2020-08-13
US20220110922A1 (en) 2022-04-14
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AU2020218185A1 (en) 2021-08-26
JP7633169B2 (ja) 2025-02-19
AU2020218185B2 (en) 2025-01-23
CA3128043A1 (en) 2020-08-13
EP3920912A1 (en) 2021-12-15
JP2022519131A (ja) 2022-03-18
HUE072626T2 (hu) 2025-11-28
US12527776B2 (en) 2026-01-20
PT3920912T (pt) 2025-09-09
ES3040555T3 (en) 2025-11-03

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