CN113557014A - 治疗有机酸血症的方法 - Google Patents

治疗有机酸血症的方法 Download PDF

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Publication number
CN113557014A
CN113557014A CN202080020521.7A CN202080020521A CN113557014A CN 113557014 A CN113557014 A CN 113557014A CN 202080020521 A CN202080020521 A CN 202080020521A CN 113557014 A CN113557014 A CN 113557014A
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CN
China
Prior art keywords
coa
pharmaceutically acceptable
acid
alkyl
compound
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CN202080020521.7A
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English (en)
Chinese (zh)
Inventor
B·沃姆霍夫
J·里尔登
R·费格勒
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Hemostatic Therapy Co ltd
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Hemostatic Therapy Co ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Hemostatic Therapy Co ltd filed Critical Hemostatic Therapy Co ltd
Publication of CN113557014A publication Critical patent/CN113557014A/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
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • 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
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • 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
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/205—Amine addition salts of organic acids; Inner quaternary ammonium salts, e.g. betaine, carnitine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive 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
    • 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)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
CN202080020521.7A 2019-03-14 2020-03-13 治疗有机酸血症的方法 Pending CN113557014A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962818372P 2019-03-14 2019-03-14
US62/818,372 2019-03-14
PCT/US2020/022760 WO2020186216A1 (en) 2019-03-14 2020-03-13 Methods of treating organic acidemias

Publications (1)

Publication Number Publication Date
CN113557014A true CN113557014A (zh) 2021-10-26

Family

ID=72426817

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080020521.7A Pending CN113557014A (zh) 2019-03-14 2020-03-13 治疗有机酸血症的方法

Country Status (11)

Country Link
US (1) US20220142955A1 (https=)
EP (1) EP3937922A4 (https=)
JP (1) JP2022525339A (https=)
KR (1) KR20210139274A (https=)
CN (1) CN113557014A (https=)
AU (1) AU2020236023A1 (https=)
BR (1) BR112021018036A2 (https=)
CA (1) CA3133260A1 (https=)
EA (1) EA202192497A1 (https=)
IL (1) IL285822A (https=)
WO (1) WO2020186216A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022216848A1 (en) * 2021-04-06 2022-10-13 Hemoshear Therapeutics, Inc. Methods of treating methylmalonic acidemia and propionic acidemia

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1856300A (zh) * 2003-07-28 2006-11-01 普里库实验室股份有限公司 治疗与代谢紊乱相关的状况的方法和材料
CN102802412A (zh) * 2009-12-08 2012-11-28 海玛奎斯特医药公司 用于治疗红细胞病症的方法及低剂量方案

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IT1249706B (it) * 1991-09-18 1995-03-09 Sigma Tau Ind Farmaceuti Uso di l-carnitina nella gravidanza a rischio.
AU3889197A (en) * 1996-07-26 1998-02-20 Douglas V Faller Compositions comprising an inducing agent and an anti-viral agent for the treat ment of blood, viral and cellular disorders
US20120289476A1 (en) * 2009-12-11 2012-11-15 Jay Allan Barth Methods for treating methylmalonic acidemia
US9018176B2 (en) * 2010-12-02 2015-04-28 Susan Perrine Inducers of hematopoiesis and fetal globin production for treatment of cytopenias and hemoglobin disorders
ES2795798T3 (es) * 2011-07-06 2020-11-24 Ptc Therapeutics Inc Tratamiento de aciduria metilmalónica, aciduria isovalérica, y otras acidurias orgánicas con tocotrienol quinonas
EP2877444B1 (en) * 2012-07-27 2020-09-02 The Broad Institute, Inc. Inhibitors of histone deacetylase
US20180064154A9 (en) * 2015-09-01 2018-03-08 D. Holmes Morton Management of propionate metabolism disorders
JO3543B1 (ar) * 2015-09-28 2020-07-05 Applied Pharma Res تراكيب حمض أميني إصدار معدل تعطى عن طريق الفم
WO2017139697A1 (en) * 2016-02-10 2017-08-17 Synlogic, Inc. Bacteria engineered to treat diseases associated with hyperammonemia
WO2017184583A1 (en) * 2016-04-19 2017-10-26 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Anaplerotic agents for treatment of disorders of propionate and long chain fat metabolism

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1856300A (zh) * 2003-07-28 2006-11-01 普里库实验室股份有限公司 治疗与代谢紊乱相关的状况的方法和材料
CN102802412A (zh) * 2009-12-08 2012-11-28 海玛奎斯特医药公司 用于治疗红细胞病症的方法及低剂量方案

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
B CHERRUAU，等: "Propionic acidemia and sodium dipropylacetate", 《C R SEANCES ACAD SCI D.》, vol. 285, no. 4 *
IN OK HWANG，等: "The First Neonatal Case of Neonatal Argininosuccinic Aciduria in Korea", 《J KOREAN SOC NEONATOL》, vol. 18, no. 1, pages 143 - 146 *

Also Published As

Publication number Publication date
IL285822A (en) 2021-10-31
EP3937922A4 (en) 2022-11-30
CA3133260A1 (en) 2020-09-17
EP3937922A1 (en) 2022-01-19
JP2022525339A (ja) 2022-05-12
US20220142955A1 (en) 2022-05-12
WO2020186216A1 (en) 2020-09-17
BR112021018036A2 (pt) 2021-11-23
KR20210139274A (ko) 2021-11-22
EA202192497A1 (ru) 2021-11-18
AU2020236023A1 (en) 2021-11-04

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