ES2795798T3 - Tratamiento de aciduria metilmalónica, aciduria isovalérica, y otras acidurias orgánicas con tocotrienol quinonas - Google Patents

Tratamiento de aciduria metilmalónica, aciduria isovalérica, y otras acidurias orgánicas con tocotrienol quinonas Download PDF

Info

Publication number
ES2795798T3
ES2795798T3 ES12807735T ES12807735T ES2795798T3 ES 2795798 T3 ES2795798 T3 ES 2795798T3 ES 12807735 T ES12807735 T ES 12807735T ES 12807735 T ES12807735 T ES 12807735T ES 2795798 T3 ES2795798 T3 ES 2795798T3
Authority
ES
Spain
Prior art keywords
compound
tocotrienol
tocotrienol quinone
aciduria
alpha
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.)
Active
Application number
ES12807735T
Other languages
English (en)
Spanish (es)
Inventor
Guy Miller
Carlo Dionisi-Vici
Enrico Bertini
Diego Martinelli
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.)
PTC Therapeutics Inc
Original Assignee
PTC Therapeutics Inc
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 PTC Therapeutics Inc filed Critical PTC Therapeutics Inc
Application granted granted Critical
Publication of ES2795798T3 publication Critical patent/ES2795798T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • 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)
ES12807735T 2011-07-06 2012-07-05 Tratamiento de aciduria metilmalónica, aciduria isovalérica, y otras acidurias orgánicas con tocotrienol quinonas Active ES2795798T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161505051P 2011-07-06 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

Publications (1)

Publication Number Publication Date
ES2795798T3 true ES2795798T3 (es) 2020-11-24

Family

ID=47437452

Family Applications (1)

Application Number Title Priority Date Filing Date
ES12807735T Active ES2795798T3 (es) 2011-07-06 2012-07-05 Tratamiento de aciduria metilmalónica, aciduria isovalérica, y otras acidurias orgánicas con tocotrienol quinonas

Country Status (6)

Country Link
US (3) US20150057363A1 (enExample)
EP (1) EP2729004B1 (enExample)
JP (3) JP6728520B2 (enExample)
ES (1) ES2795798T3 (enExample)
PT (1) PT2729004T (enExample)
WO (1) WO2013006737A1 (enExample)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2580584C (en) 2003-09-19 2015-07-28 Galileo Pharmaceuticals, Inc. Use of alpha-tocotrienol for treatment of mitochondrial diseases
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
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

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB0204232D0 (en) 2002-02-22 2002-04-10 Isis Innovation Assay
EP1378753B1 (en) 2002-07-01 2006-05-31 Santhera Pharmaceuticals (Schweiz) GmbH A screening method and compounds for treating Friedreich Ataxia
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

Also Published As

Publication number Publication date
US20180303770A1 (en) 2018-10-25
JP2020029465A (ja) 2020-02-27
JP2014528914A (ja) 2014-10-30
US20150057363A1 (en) 2015-02-26
JP2018030892A (ja) 2018-03-01
WO2013006737A1 (en) 2013-01-10
JP6728520B2 (ja) 2020-07-22
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

Similar Documents

Publication Publication Date Title
US20210145769A1 (en) Treatment of methylmalonic aciduria, isovaleric aciduria, and other organic acidurias with tocotrienol quinones
US20220133648A1 (en) Treatment of leigh syndrome and leigh-like syndrome with tocotrienol quinones
WO2013006736A1 (en) Treatment of leigh syndrome and leigh-like syndrome, including complications of sucla2 mutations, with tocotrienol quinones
US20130116336A1 (en) Treatment of ataxia telangiectasia
Payen et al. Muscular metabolism during oxygen supplementation in patients with chronic hypoxemia
ES2823728T3 (es) Variantes de cola de agentes terapéuticos con actividad redox para el tratamiento de enfermedades mitocondriales y otras afecciones y la modulación de biomarcadores de energía
US10195161B2 (en) Treatment of leber's hereditary optic neuropathy and dominant optic atrophy with tocotrienol quinones
WO2012170773A1 (en) Adjunctive therapy for the treatment of mitochondrial disorders with quinones and naphthoquinones
JP2013538799A (ja) ビタミンkを用いたミトコンドリア病の処置
US20100273892A1 (en) Formulations of tocotrienol quinones for the treatment of ophthalmic diseases
TW201605434A (zh) 使用半胱胺及其衍生物治療粒腺體疾病
KR20190133206A (ko) 미토콘드리아 질환의 신규 치료
EP3043788B1 (en) D-glyceric acid or dl-glyceric acid for use in the treatment of degeneration diseases related to aging
US11331319B2 (en) Combination treatment for neuropsychiatric disorders
Fukunaga et al. Steady‐state pharmacokinetics of zonisamide in plasma, whole blood, and erythrocytes in dogs
PT2229943E (pt) Composto para ser utilizado no tratamento das neuropatias periféricas.
HK1168028B (en) Treatment of leber's hereditary optic neuropathy and dominant optic atrophy with tocotrienol quinones