JP2022525339A - 有機酸血症の治療方法 - Google Patents

有機酸血症の治療方法 Download PDF

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JP2022525339A
JP2022525339A JP2021555384A JP2021555384A JP2022525339A JP 2022525339 A JP2022525339 A JP 2022525339A JP 2021555384 A JP2021555384 A JP 2021555384A JP 2021555384 A JP2021555384 A JP 2021555384A JP 2022525339 A JP2022525339 A JP 2022525339A
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coa
hours
alkyl
acidemia
acid
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JP2022525339A5 (https=
Inventor
ワムホフ ブライアン
リアードン ジョン
フィグラー ロバート
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ヘモシアー セラピューティクス，インコーポレイティド
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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
    • 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
    • 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)
JP2021555384A 2019-03-14 2020-03-13 有機酸血症の治療方法 Pending JP2022525339A (ja)

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 (2)

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JP2022525339A true JP2022525339A (ja) 2022-05-12
JP2022525339A5 JP2022525339A5 (https=) 2023-03-24

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ID=72426817

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JP2021555384A Pending JP2022525339A (ja) 2019-03-14 2020-03-13 有機酸血症の治療方法

Country Status (11)

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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 (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH05194206A (ja) * 1991-09-18 1993-08-03 Sigma Tau Ind Farmaceut Riunite Spa 妊娠におけるｌ−カルニチンの使用
JP2001527517A (ja) * 1996-07-26 2001-12-25 スーザン・ピー・ペリーネ 血液、ウイルス性および細胞性疾患の治療組成物
WO2011072281A1 (en) * 2009-12-11 2011-06-16 Ptc Therapeutics, Inc. Methods for treating methylmalonic acidemia
JP2014528914A (ja) * 2011-07-06 2014-10-30 エジソン ファーマシューティカルズ， インコーポレイテッド メチルマロン酸尿症、イソ吉草酸尿症、および他の有機酸尿症のトコトリエノールキノンによる処置
US20170055562A1 (en) * 2015-09-01 2017-03-02 D. Holmes Morton Management of propionate metabolism disorders
US20180185311A1 (en) * 2015-09-28 2018-07-05 Apr Applied Pharma Research S.A. Modified release orally administered amino acid formulations

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050027006A1 (en) * 2003-07-28 2005-02-03 Reuben Matalon Methods and materials for treating conditions associated with metabolic disorders
WO2011072086A1 (en) * 2009-12-08 2011-06-16 Hemaquest Pharmaceuticals, Inc. Methods and low dose regimens for treating red blood cell disorders
US9018176B2 (en) * 2010-12-02 2015-04-28 Susan Perrine Inducers of hematopoiesis and fetal globin production for treatment of cytopenias and hemoglobin disorders
EP2877444B1 (en) * 2012-07-27 2020-09-02 The Broad Institute, Inc. Inhibitors of histone deacetylase
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 (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH05194206A (ja) * 1991-09-18 1993-08-03 Sigma Tau Ind Farmaceut Riunite Spa 妊娠におけるｌ−カルニチンの使用
JP2001527517A (ja) * 1996-07-26 2001-12-25 スーザン・ピー・ペリーネ 血液、ウイルス性および細胞性疾患の治療組成物
WO2011072281A1 (en) * 2009-12-11 2011-06-16 Ptc Therapeutics, Inc. Methods for treating methylmalonic acidemia
JP2014528914A (ja) * 2011-07-06 2014-10-30 エジソン ファーマシューティカルズ， インコーポレイテッド メチルマロン酸尿症、イソ吉草酸尿症、および他の有機酸尿症のトコトリエノールキノンによる処置
US20170055562A1 (en) * 2015-09-01 2017-03-02 D. Holmes Morton Management of propionate metabolism disorders
US20180185311A1 (en) * 2015-09-28 2018-07-05 Apr Applied Pharma Research S.A. Modified release orally administered amino acid formulations

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
COMPTES RENDUS DES SEANCES DE I'ACADEMIE DES SCIENCES. SERIE D, SICENCES NATURELLES, vol. 289, no. 5, JPN6024007296, 1979, pages 469 - 472, ISSN: 0005270080 *
JOURNAL OF THE KOREAN SOCIETY OF NEONATOLOGY, vol. 18, JPN6024007297, 2011, pages 143 - 137, ISSN: 0005270079 *
小児内科, vol. 33, no. 7, JPN6024007295, 2011, pages 964 - 1036, ISSN: 0005270081 *

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
US20220142955A1 (en) 2022-05-12
WO2020186216A1 (en) 2020-09-17
CN113557014A (zh) 2021-10-26
BR112021018036A2 (pt) 2021-11-23
KR20210139274A (ko) 2021-11-22
EA202192497A1 (ru) 2021-11-18
AU2020236023A1 (en) 2021-11-04

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