JP6728520B2 - 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 - Google Patents

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 Download PDF

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JP6728520B2
JP6728520B2 JP2014519307A JP2014519307A JP6728520B2 JP 6728520 B2 JP6728520 B2 JP 6728520B2 JP 2014519307 A JP2014519307 A JP 2014519307A JP 2014519307 A JP2014519307 A JP 2014519307A JP 6728520 B2 JP6728520 B2 JP 6728520B2
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tocotrienol quinone
tocotrienol
aciduria
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quinone
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ガイ エム. ミラー,
ガイ エム. ミラー,
カルロ ディニーシ−ヴーチ,
カルロ ディニーシ−ヴーチ,
エンリーコ ベルティーニ,
エンリーコ ベルティーニ,
ディエゴ マルティネッリ,
ディエゴ マルティネッリ,
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ピーティーシー セラピューティクス, インコーポレイテッド
ピーティーシー セラピューティクス, インコーポレイテッド
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/12Ketones
    • A61K31/122Ketones having the oxygen directly attached to a ring, e.g. quinones, vitamin K1, anthralin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism

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  • Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
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  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
JP2014519307A 2011-07-06 2012-07-05 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 Expired - Fee Related JP6728520B2 (ja)

Applications Claiming Priority (3)

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US201161505051P 2011-07-06 2011-07-06
US61/505,051 2011-07-06
PCT/US2012/045628 WO2013006737A1 (en) 2011-07-06 2012-07-05 Treatment of methylmalonic aciduria, isovaleric aciduria, and other organic acidurias with tocotrienol quinones

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JP2017231924A Division JP2018030892A (ja) 2011-07-06 2017-12-01 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
JP2019215845A Division JP2020029465A (ja) 2011-07-06 2019-11-28 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

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JP2014528914A JP2014528914A (ja) 2014-10-30
JP2014528914A5 JP2014528914A5 (enExample) 2015-09-03
JP6728520B2 true JP6728520B2 (ja) 2020-07-22

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JP2014519307A Expired - Fee Related JP6728520B2 (ja) 2011-07-06 2012-07-05 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
JP2017231924A Pending JP2018030892A (ja) 2011-07-06 2017-12-01 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
JP2019215845A Withdrawn JP2020029465A (ja) 2011-07-06 2019-11-28 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

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JP2019215845A Withdrawn JP2020029465A (ja) 2011-07-06 2019-11-28 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

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US (3) US20150057363A1 (enExample)
EP (1) EP2729004B1 (enExample)
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ES (1) ES2795798T3 (enExample)
PT (1) PT2729004T (enExample)
WO (1) WO2013006737A1 (enExample)

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EA019675B1 (ru) 2006-02-22 2014-05-30 Эдисон Фармасьютикалз, Инк. Редокс-активные терапевтические средства для лечения митохондриальных заболеваний и модуляции биомаркера коэнзима q
EP3018122B1 (en) 2007-11-06 2018-10-10 BioElectron Technology Corporation 4-(p-quinonyl)-2-hydroxybutanamide derivatives for treatment of mitochondrial diseases
CA2708961C (en) 2008-01-08 2017-03-28 Ptc Therapeutics, Inc. (het)aryl-p-quinone derivatives for treatment of mitochondrial diseases
WO2009111576A2 (en) 2008-03-05 2009-09-11 Edison Pharmaceuticals, Inc. 2-SUBSTITUTED-p-QUINONE DERIVATIVES FOR TREATMENT OF OXIDATIVE STRESS DISEASES
WO2009158348A1 (en) 2008-06-25 2009-12-30 Edison Pharmaceuticals, Inc. 2-heterocyclylaminoalkyl-(p-quinone) derivatives for treatment of oxidative stress diseases
BRPI0918139B1 (pt) 2008-09-10 2024-04-30 Ptc Therapeutics, Inc Composição, alimento medicinal ou funcional, suplemento alimentar ou dietético para tratamento de distúrbios de desenvolvimento pervasivos com terapêuticos redox-ativos
CA2740773A1 (en) 2008-10-14 2010-04-22 Edison Pharmaceuticals, Inc. Treatment of oxidative stress disorders including contrast nephropathy, radiation damage and disruptions in the function of red cells
CN102264730B (zh) 2008-10-28 2016-04-13 爱迪生药物公司 用于生产生育三烯酚及衍生物的方法
JP2012525399A (ja) 2009-04-28 2012-10-22 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 トコトリエノールキノンを用いる、レーバー遺伝性視神経症および優性遺伝性視神経萎縮症の治療
WO2012174286A1 (en) 2011-06-14 2012-12-20 Edison Pharmaceuticals, Inc. Catechol derivatives for treatment of oxidative stress diseases
JP2014520894A (ja) 2011-07-19 2014-08-25 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 非アルファトコトリエノールの存在下でのアルファトコトリエノールの選択的酸化のための方法
CA2883879A1 (en) 2012-09-07 2014-03-13 Edison Pharmaceuticals, Inc. Quinone derivatives for use in the modulation of redox status of individuals
US9670170B2 (en) 2013-03-15 2017-06-06 Bioelectron Technology Corporation Resorufin derivatives for treatment of oxidative stress disorders
CA2906145A1 (en) 2013-03-15 2014-09-18 Ptc Therapeutics, Inc. Alkyl-heteroaryl substituted quinone derivatives for treatment of oxidative stress disorders
US9868711B2 (en) 2013-03-15 2018-01-16 Bioelectron Technology Corporation Phenazine-3-one and phenothiazine-3-one derivatives for treatment of oxidative stress disorders
US9296712B2 (en) 2013-03-15 2016-03-29 Edison Pharmaceuticals, Inc. Resorufin derivatives for treatment of oxidative stress disorders
WO2016100579A1 (en) 2014-12-16 2016-06-23 Edison Pharmaceuticals, Inc., Polymorphic and amorphous forms of (r)-2-hydroxy-2-methyl-4-(2,4,5-trimethyl-3,6-dioxocyclohexa-1,4-dienyl)butanamide
WO2017106786A1 (en) 2015-12-16 2017-06-22 Bioelectron Technology Corporation Improved methods for enriching alpha-tocotrienol from mixed tocol compositions
CN113024369A (zh) 2015-12-17 2021-06-25 Ptc医疗公司 用于治疗氧化应急障碍的化合物
US11083702B2 (en) 2016-04-19 2021-08-10 University of Pittsburgh—of the Commonwealth System of Higher Education Anaplerotic agents for treatment of disorders of propionate and long chain fat metabolism
JP2018083799A (ja) 2016-11-15 2018-05-31 バイオエレクトロン テクノロジー コーポレイション 2−置換アミノ−ナフト[1,2−d]イミダゾール−5−オン化合物またはその製薬学上許容される塩
JP7586815B2 (ja) 2018-10-17 2024-11-19 ピーティーシー セラピューティクス, インコーポレイテッド α-シヌクレイノパチー、タウオパチー、および他の障害を抑制および処置するための2,3,5-トリメチル-6-ノニルシクロヘキサ-2,5-ジエン-1,4-ジオン
AU2020236023A1 (en) * 2019-03-14 2021-11-04 Hemoshear Therapeutics, Inc. Methods of treating organic acidemias
CN114364664A (zh) * 2019-07-09 2022-04-15 伊卡根有限责任公司 Bcat调节
CN118175991A (zh) 2021-07-08 2024-06-11 Ptc医疗公司 包含2,3,5-三甲基-6-壬基环己-2,5-二烯-1,4-二酮的药物组合物

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US20080187911A1 (en) * 2003-01-30 2008-08-07 Research Development Foundation Methods and compositions for analysis of mitochondrial-related gene expression
CA2610152C (en) * 2005-06-01 2018-04-10 Edison Pharmaceuticals, Inc. Redox-active therapeutics for treatment of mitochondrial diseases and other conditions and modulation of energy biomarkers
EP1749552A1 (en) * 2005-07-29 2007-02-07 Neuropharma S.A. Use of collismycin and derivatives thereof as oxidative stress inhibitors
JP2012525399A (ja) * 2009-04-28 2012-10-22 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 トコトリエノールキノンを用いる、レーバー遺伝性視神経症および優性遺伝性視神経萎縮症の治療
MX2011011310A (es) * 2009-04-28 2011-11-18 Edison Pharmaceuticals Inc Formulacion de tocotrienol quinonas para el tratamiento de enfermedades oftalmicas.
WO2011072281A1 (en) * 2009-12-11 2011-06-16 Ptc Therapeutics, Inc. Methods for treating methylmalonic acidemia

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US20180303770A1 (en) 2018-10-25
JP2020029465A (ja) 2020-02-27
ES2795798T3 (es) 2020-11-24
JP2014528914A (ja) 2014-10-30
US20150057363A1 (en) 2015-02-26
JP2018030892A (ja) 2018-03-01
WO2013006737A1 (en) 2013-01-10
US20210145769A1 (en) 2021-05-20
EP2729004A1 (en) 2014-05-14
PT2729004T (pt) 2020-06-08
EP2729004B1 (en) 2020-03-25
EP2729004A4 (en) 2014-11-19

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