CN108348527A - 用于治疗癫痫的方法 - Google Patents

用于治疗癫痫的方法 Download PDF

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Publication number
CN108348527A
CN108348527A CN201680063273.8A CN201680063273A CN108348527A CN 108348527 A CN108348527 A CN 108348527A CN 201680063273 A CN201680063273 A CN 201680063273A CN 108348527 A CN108348527 A CN 108348527A
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CN
China
Prior art keywords
nonsense mutation
epilepsy
patient
visit
cdkl5
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CN201680063273.8A
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English (en)
Chinese (zh)
Inventor
埃伦·韦尔奇
富泽友希
普里亚·瓦齐拉尼
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PTC Therapeutics Inc
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PTC Therapeutics Inc
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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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/4245—Oxadiazoles
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants

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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)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Pain & Pain Management (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
CN201680063273.8A 2015-10-30 2016-10-28 用于治疗癫痫的方法 Pending CN108348527A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562248891P 2015-10-30 2015-10-30
US62/248,891 2015-10-30
PCT/US2016/059262 WO2017075312A1 (en) 2015-10-30 2016-10-28 Methods for treating epilepsy

Publications (1)

Publication Number Publication Date
CN108348527A true CN108348527A (zh) 2018-07-31

Family

ID=58632013

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201680063273.8A Pending CN108348527A (zh) 2015-10-30 2016-10-28 用于治疗癫痫的方法

Country Status (15)

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US (2) US10517853B2 (enExample)
EP (1) EP3368042A4 (enExample)
JP (1) JP2018531975A (enExample)
KR (1) KR20180081516A (enExample)
CN (1) CN108348527A (enExample)
AR (1) AR106530A1 (enExample)
BR (1) BR112018008838A8 (enExample)
CA (1) CA3003132A1 (enExample)
CO (1) CO2018004684A2 (enExample)
HK (1) HK1251965A1 (enExample)
IL (1) IL258988A (enExample)
MX (1) MX2018005361A (enExample)
PH (1) PH12018500885A1 (enExample)
TW (1) TW201729807A (enExample)
WO (1) WO2017075312A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117987538A (zh) * 2024-04-07 2024-05-07 北京诺禾致源科技股份有限公司 用于检测depdc5突变基因的试剂在制备诊断或预测家族性局灶性癫痫伴可变灶1型的试剂盒中的应用

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CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
CN116059236A (zh) 2015-12-14 2023-05-05 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
KR20260009385A (ko) 2017-08-25 2026-01-19 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
US11301527B2 (en) * 2019-05-06 2022-04-12 Acto Technologies Inc. Event-driven content recommendation engine
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
US12624011B2 (en) * 2020-03-17 2026-05-12 Sumitomo Pharma Co., Ltd. Oxadiazole derivative
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases

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US20100088778A1 (en) * 2005-06-16 2010-04-08 John Charles Mulley Methods of Treatment, and Diagnosis of Epilepsy by Detecting Mutations in the SCN1A Gene
CN103189060A (zh) * 2010-08-05 2013-07-03 里尔法律与医疗卫生第二大学 用于对无义突变介导的疾病进行治疗的化合物和含有所述化合物的药物组合物

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Cited By (1)

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Publication number Priority date Publication date Assignee Title
CN117987538A (zh) * 2024-04-07 2024-05-07 北京诺禾致源科技股份有限公司 用于检测depdc5突变基因的试剂在制备诊断或预测家族性局灶性癫痫伴可变灶1型的试剂盒中的应用

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KR20180081516A (ko) 2018-07-16
US20200188362A1 (en) 2020-06-18
MX2018005361A (es) 2018-06-07
US20180333397A1 (en) 2018-11-22
JP2018531975A (ja) 2018-11-01
EP3368042A4 (en) 2019-06-26
BR112018008838A2 (pt) 2018-11-06
BR112018008838A8 (pt) 2019-02-26
EP3368042A1 (en) 2018-09-05
TW201729807A (zh) 2017-09-01
CA3003132A1 (en) 2017-05-04
CO2018004684A2 (es) 2018-08-10
US10517853B2 (en) 2019-12-31
PH12018500885A1 (en) 2018-10-29
WO2017075312A1 (en) 2017-05-04
HK1251965A1 (zh) 2019-05-10
AR106530A1 (es) 2018-01-24
IL258988A (en) 2018-06-28

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