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
weight
quinone
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JP2014528914A (ja
JP2014528914A5 (OSRAM
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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，
Original Assignee
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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    • 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
    • A61K31/12—Ketones
    • A61K31/122—Ketones having the oxygen directly attached to a ring, e.g. quinones, vitamin K1, anthralin
    • 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
    • A61K31/045—Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates
    • A61K31/05—Phenols
    • 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

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (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)

Application Number Priority Date Filing Date Title
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 (OSRAM) 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 (OSRAM)
EP (1) EP2729004B1 (OSRAM)
JP (3) JP6728520B2 (OSRAM)
ES (1) ES2795798T3 (OSRAM)
PT (1) PT2729004T (OSRAM)
WO (1) WO2013006737A1 (OSRAM)

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JP5374162B2 (ja) 2006-02-22 2013-12-25 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 ミトコンドリア病および他の症状の処置のためのレドックス活性化治療の側鎖変異体およびエネルギーバイオマーカーの調節
EP3456707B1 (en) 2007-11-06 2020-04-15 PTC Therapeutics, Inc. 4- (p-quinonyl)-2-hydroxybutanamide derivatives for treatment of mitochondrial diseases
EA023618B1 (ru) 2008-01-08 2016-06-30 Эдисон Фармасьютикалз, Инк. Производные (гет)арил-п-хинона для лечения митохондриальных болезней
CA2717734A1 (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
EP3827815B1 (en) 2008-09-10 2023-09-06 PTC Therapeutics, Inc. Treatment of pervasive developmental disorders with redox-active therapeutics
WO2010045220A1 (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
TR201819154T4 (tr) 2008-10-28 2019-01-21 Bioelectron Tech Corp Alfa-tokotrienol kuinone ve ara ürünlerini içeren bileşim.
WO2010126911A1 (en) 2009-04-28 2010-11-04 Edison Pharmaceuticals, Inc. Treatment of leber's hereditary optic neuropathy and dominant optic atrophy with tocotrienol quinones
US9464016B2 (en) 2011-06-14 2016-10-11 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
CA2883882A1 (en) 2012-09-07 2014-03-13 Edison Pharmaceuticals, Inc. Benzoquinone derivatives for treating 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
EP2970158B1 (en) 2013-03-15 2019-02-20 BioElectron Technology Corporation Alkyl-heteroaryl substituted quinone 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
US9670170B2 (en) 2013-03-15 2017-06-06 Bioelectron Technology Corporation Resorufin derivatives for treatment of oxidative stress disorders
EP3233786B1 (en) 2014-12-16 2022-03-16 PTC Therapeutics, Inc. Polymorphic and amorphous forms of (r)-2-hydroxy-2-methyl-4-(2,4,5-trimethyl-3,6-dioxocyclohexa-1,4-dienyl)butanamide
EP3390377A1 (en) 2015-12-16 2018-10-24 BioElectron Technology Corporation Improved methods for enriching alpha-tocotrienol from mixed tocol compositions
US10703701B2 (en) 2015-12-17 2020-07-07 Ptc Therapeutics, Inc. Fluoroalkyl, fluoroalkoxy, phenoxy, heteroaryloxy, alkoxy, and amine 1,4-benzoquinone derivatives for treatment of oxidative stress disorders
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
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
PL3866772T3 (pl) 2018-10-17 2024-04-08 Ptc Therapeutics, Inc. 2,3,5-trimetylo-6-nonylocykloheksa-2,5-dieno-1,4-dion do hamowania i leczenia alfa-synukleinopatii, tauopatii i innych zaburzeń
WO2020186216A1 (en) * 2019-03-14 2020-09-17 Hemoshear Therapeutics, Llc 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
KR20240032997A (ko) 2021-07-08 2024-03-12 피티씨 테라퓨틱스, 인크. 2,3,5-트리메틸-6-노닐사이클로헥사-2,5-디엔-1,4-디온을 포함하는 약제학적 조성물

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GB0204232D0 (en) 2002-02-22 2002-04-10 Isis Innovation Assay
ATE328283T1 (de) 2002-07-01 2006-06-15 Santhera Pharmaceuticals Ch Screeningverfahren und verbindungen zur behandlung von friedreich ataxia
US20080187911A1 (en) * 2003-01-30 2008-08-07 Research Development Foundation Methods and compositions for analysis of mitochondrial-related gene expression
WO2006130775A2 (en) * 2005-06-01 2006-12-07 Edison Pharmaceuticals, Inc. Redox-active therapeutics for treatment of mitochondrial diseases and other conditions and modulation of energy biomakers
EP1749552A1 (en) * 2005-07-29 2007-02-07 Neuropharma S.A. Use of collismycin and derivatives thereof as oxidative stress inhibitors
WO2010126911A1 (en) * 2009-04-28 2010-11-04 Edison Pharmaceuticals, Inc. Treatment of leber's hereditary optic neuropathy and dominant optic atrophy with tocotrienol quinones
WO2010126909A1 (en) * 2009-04-28 2010-11-04 Edison Pharmaceuticals, Inc. Formulation of tocotrienol quinones for the treatment of ophthalmic diseases
WO2011072281A1 (en) * 2009-12-11 2011-06-16 Ptc Therapeutics, Inc. Methods for treating methylmalonic acidemia

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

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