KR20240138572A - 혈우병 환자에서 환자 보고 결과를 개선하는 데 사용하기 위한 피투시란 - Google Patents

혈우병 환자에서 환자 보고 결과를 개선하는 데 사용하기 위한 피투시란 Download PDF

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Publication number
KR20240138572A
KR20240138572A KR1020247024462A KR20247024462A KR20240138572A KR 20240138572 A KR20240138572 A KR 20240138572A KR 1020247024462 A KR1020247024462 A KR 1020247024462A KR 20247024462 A KR20247024462 A KR 20247024462A KR 20240138572 A KR20240138572 A KR 20240138572A
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KR
South Korea
Prior art keywords
qol
patient
pitusiran
hemophilia
questionnaire
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Pending
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KR1020247024462A
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English (en)
Korean (ko)
Inventor
샤우나 앤더슨
조지 바르텔트-호퍼
프로나베쉬 다스마하파트라
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젠자임 코포레이션
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Publication of KR20240138572A publication Critical patent/KR20240138572A/ko
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
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/7056—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing five-membered rings with nitrogen as a ring hetero atom
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020247024462A 2021-12-22 2022-12-22 혈우병 환자에서 환자 보고 결과를 개선하는 데 사용하기 위한 피투시란 Pending KR20240138572A (ko)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US202163293070P 2021-12-22 2021-12-22
US63/293,070 2021-12-22
US202263305550P 2022-02-01 2022-02-01
US63/305,550 2022-02-01
US202263335535P 2022-04-27 2022-04-27
US63/335,535 2022-04-27
US202263359717P 2022-07-08 2022-07-08
US63/359,717 2022-07-08
US202263382223P 2022-11-03 2022-11-03
US63/382,223 2022-11-03
US202263386489P 2022-12-07 2022-12-07
US63/386,489 2022-12-07
PCT/US2022/082213 WO2023122713A1 (en) 2021-12-22 2022-12-22 Fitusiran for use in improving patient-reported outcome in hemophilia patients

Publications (1)

Publication Number Publication Date
KR20240138572A true KR20240138572A (ko) 2024-09-20

Family

ID=85227438

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247024462A Pending KR20240138572A (ko) 2021-12-22 2022-12-22 혈우병 환자에서 환자 보고 결과를 개선하는 데 사용하기 위한 피투시란

Country Status (10)

Country Link
US (1) US20250064842A1 (https=)
EP (1) EP4452280A1 (https=)
JP (1) JP2025500412A (https=)
KR (1) KR20240138572A (https=)
AU (1) AU2022419588A1 (https=)
CA (1) CA3243297A1 (https=)
IL (1) IL313677A (https=)
MX (1) MX2024007894A (https=)
TW (1) TW202342065A (https=)
WO (1) WO2023122713A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025171279A1 (en) * 2024-02-09 2025-08-14 Genzyme Corporation Treatment of hemophilia with fitusiran

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
KR20180095843A (ko) 2015-12-07 2018-08-28 젠자임 코포레이션 Serpinc1-연관 장애의 치료를 위한 방법 및 조성물
TW202434265A (zh) 2017-07-10 2024-09-01 美商健贊公司 於患有血友病之個體中治療出血事件之方法及組成物
WO2022120291A1 (en) * 2020-12-06 2022-06-09 Genzyme Corporation Treatment of hemophilia with fitusiran

Also Published As

Publication number Publication date
TW202342065A (zh) 2023-11-01
JP2025500412A (ja) 2025-01-09
AU2022419588A1 (en) 2024-08-01
MX2024007894A (es) 2024-08-20
US20250064842A1 (en) 2025-02-27
EP4452280A1 (en) 2024-10-30
WO2023122713A1 (en) 2023-06-29
IL313677A (en) 2024-08-01
CA3243297A1 (en) 2023-06-29

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