JP2018521993A5 - - Google Patents

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Publication number
JP2018521993A5
JP2018521993A5 JP2017566000A JP2017566000A JP2018521993A5 JP 2018521993 A5 JP2018521993 A5 JP 2018521993A5 JP 2017566000 A JP2017566000 A JP 2017566000A JP 2017566000 A JP2017566000 A JP 2017566000A JP 2018521993 A5 JP2018521993 A5 JP 2018521993A5
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Japan
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day
effective amount
therapeutically effective
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JP2017566000A
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Japanese (ja)
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JP6599484B2 (ja
JP2018521993A (ja
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Priority claimed from PCT/US2016/038110 external-priority patent/WO2016205671A1/en
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JP2017566000A 2015-06-17 2016-06-17 ミトコンドリアdna枯渇症候群を含む不均衡なヌクレオチドプールによって引き起こされる疾患のためのデオキシヌクレオシド療法 Active JP6599484B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562180914P 2015-06-17 2015-06-17
US62/180,914 2015-06-17
PCT/US2016/038110 WO2016205671A1 (en) 2015-06-17 2016-06-17 Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes

Related Child Applications (2)

Application Number Title Priority Date Filing Date
JP2019182192A Division JP7036782B2 (ja) 2015-06-17 2019-10-02 ミトコンドリアdna枯渇症候群を含む不均衡なヌクレオチドプールによって引き起こされる疾患のためのデオキシヌクレオシド療法
JP2019182191A Division JP6675037B2 (ja) 2015-06-17 2019-10-02 ミトコンドリアdna枯渇症候群を含む不均衡なヌクレオチドプールによって引き起こされる疾患のためのデオキシヌクレオシド療法

Publications (3)

Publication Number Publication Date
JP2018521993A JP2018521993A (ja) 2018-08-09
JP2018521993A5 true JP2018521993A5 (enExample) 2019-07-04
JP6599484B2 JP6599484B2 (ja) 2019-10-30

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ID=57546446

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2017566000A Active JP6599484B2 (ja) 2015-06-17 2016-06-17 ミトコンドリアdna枯渇症候群を含む不均衡なヌクレオチドプールによって引き起こされる疾患のためのデオキシヌクレオシド療法
JP2019182191A Active JP6675037B2 (ja) 2015-06-17 2019-10-02 ミトコンドリアdna枯渇症候群を含む不均衡なヌクレオチドプールによって引き起こされる疾患のためのデオキシヌクレオシド療法
JP2019182192A Active JP7036782B2 (ja) 2015-06-17 2019-10-02 ミトコンドリアdna枯渇症候群を含む不均衡なヌクレオチドプールによって引き起こされる疾患のためのデオキシヌクレオシド療法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2019182191A Active JP6675037B2 (ja) 2015-06-17 2019-10-02 ミトコンドリアdna枯渇症候群を含む不均衡なヌクレオチドプールによって引き起こされる疾患のためのデオキシヌクレオシド療法
JP2019182192A Active JP7036782B2 (ja) 2015-06-17 2019-10-02 ミトコンドリアdna枯渇症候群を含む不均衡なヌクレオチドプールによって引き起こされる疾患のためのデオキシヌクレオシド療法

Country Status (24)

Country Link
US (5) US10471087B2 (enExample)
EP (3) EP3505174B1 (enExample)
JP (3) JP6599484B2 (enExample)
KR (3) KR20180039624A (enExample)
CN (2) CN116726035A (enExample)
AU (3) AU2016280293B2 (enExample)
BR (2) BR122020021913B1 (enExample)
CA (1) CA2989653A1 (enExample)
CY (2) CY1122605T1 (enExample)
DK (2) DK3310362T3 (enExample)
ES (2) ES2748556T3 (enExample)
HK (1) HK1252133A1 (enExample)
HR (2) HRP20191794T1 (enExample)
HU (2) HUE046399T2 (enExample)
IL (3) IL275256B2 (enExample)
LT (2) LT3505174T (enExample)
MX (2) MX2017016425A (enExample)
PL (2) PL3310362T3 (enExample)
PT (2) PT3505174T (enExample)
RS (2) RS60572B1 (enExample)
RU (1) RU2721492C2 (enExample)
SI (2) SI3310362T1 (enExample)
SM (2) SMT202000399T1 (enExample)
WO (1) WO2016205671A1 (enExample)

Families Citing this family (22)

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Publication number Priority date Publication date Assignee Title
US9549909B2 (en) 2013-05-03 2017-01-24 The Katholieke Universiteit Leuven Method for the treatment of dravet syndrome
US10292996B2 (en) * 2015-03-26 2019-05-21 The Trustees Of Columbia University In The City Of New York Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome
US10471087B2 (en) * 2015-06-17 2019-11-12 The Trustees Of Columbia University In The City Of New York Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial DNA depletion syndromes
EP3393470B1 (en) 2015-12-22 2021-01-20 Zogenix International Limited Metabolism resistant fenfluramine analogs and methods of using the same
CA3007286A1 (en) 2015-12-22 2017-06-29 Zogenix International Limited Fenfluramine compositions and methods of preparing the same
MX2019001799A (es) 2016-08-24 2019-06-13 Zogenix International Ltd Formulacion para inhibir la formacion de agonistas de 5-ht2b y metodos de utilizacion de la misma.
WO2019051097A1 (en) 2017-09-08 2019-03-14 The Regents Of The University Of California RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME
US10682317B2 (en) 2017-09-26 2020-06-16 Zogenix International Limited Ketogenic diet compatible fenfluramine formulation
WO2019200340A1 (en) 2018-04-12 2019-10-17 Modis Therapeutics Inc. Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools
MX2020010994A (es) 2018-04-18 2021-01-08 Univ Columbia Terapia de gen para enfermedades causadas por combinaciones desequilibradas de nucleótidos que incluyen síndromes de agotamiento de adn mitocondrial.
WO2019216919A1 (en) 2018-05-11 2019-11-14 Zogenix International Limited Compositions and methods for treating seizure-induced sudden death
EA202191079A1 (ru) 2018-11-19 2021-09-20 Зодженикс Интернэшнл Лимитед Способы лечения синдрома ретта с применением фенфлурамина
EP4017502A4 (en) * 2019-08-19 2023-11-01 Modis Therapeutics Inc. POLYMORPH FORMS OF DEOXYCYTIDINE, COMPOSITIONS COMPRISING THEM AND USES
CN114340641B (zh) * 2019-09-05 2025-04-04 米托瑞因博治疗股份有限公司 治疗线粒体dna缺失障碍
WO2021188840A1 (en) * 2020-03-19 2021-09-23 Rewrite Therapeutics, Inc. Methods and compositions for directed genome editing
US11612574B2 (en) 2020-07-17 2023-03-28 Zogenix International Limited Method of treating patients infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2)
US20220096514A1 (en) * 2020-09-29 2022-03-31 Zogenix International Limited Methods of treating thymidine kinase 2 deficiency by administering deoxycytidine and deoxythymidine
WO2022145439A1 (ja) * 2020-12-28 2022-07-07 ヤマサ醤油株式会社 筋萎縮抑制剤及び筋萎縮を抑制させる方法
CN117120060A (zh) * 2021-03-26 2023-11-24 周格尼克斯-莫迪斯公司 含有嘌呤和嘧啶的水溶液及其用途
EP4355336A4 (en) * 2021-06-18 2025-05-07 UCB Biosciences, Inc. DEOXYNUCLEOSIDE PRODRUGS FOR THE TREATMENT OF MITOCHONDRIAL DISEASES CAUSED BY UNBALANCED NUCLEOTIDE GROUPS
EP4554596A1 (en) * 2022-07-12 2025-05-21 UCB Biosciences, Inc. Methods for increasing the bioavailability of nucleoside medicinal agents
WO2024206595A2 (en) * 2023-03-30 2024-10-03 The Trustees Of Columbia University In The City Of New York Pharmacological therapy for mitochondrial dna depletion deletions syndrome involving mutations in the guk1 gene

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1911237B (zh) * 1998-08-10 2010-09-15 艾丹尼克斯(开曼)有限公司 用于治疗乙型肝炎的β-L-2'-脱氧-核苷
KR100879559B1 (ko) * 1999-02-23 2009-01-22 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 미토콘드리아 장애의 치료 또는 예방용 제약조성물
US6596700B2 (en) * 2000-05-26 2003-07-22 Idenix Pharmaceuticals Inc. Methods of treating hepatitis delta virus infection with β-L-2'-deoxy-nucleosides
WO2012125848A2 (en) * 2011-03-16 2012-09-20 Baylor College Of Medicine A method for comprehensive sequence analysis using deep sequencing technology
US10292996B2 (en) 2015-03-26 2019-05-21 The Trustees Of Columbia University In The City Of New York Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome
PL3302499T3 (pl) * 2015-06-05 2023-07-24 Fundació Hospital Universitari Vall D'hebron - Institut De Recerca Leczenie chorób mitochondrialnych
US10471087B2 (en) * 2015-06-17 2019-11-12 The Trustees Of Columbia University In The City Of New York Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial DNA depletion syndromes

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