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AGENT FOR TREATMENT OR PREVENTION OF MUSCULAR-DYSTROPHY-RELATED CARDIOMYOPATHY NEW

外国特許コード F190009982
整理番号 (S2018-0451-N0)
掲載日 2019年10月28日
出願国 世界知的所有権機関(WIPO)
国際出願番号 2019JP009742
国際公開番号 WO 2019176864
国際出願日 平成31年3月11日(2019.3.11)
国際公開日 令和元年9月19日(2019.9.19)
優先権データ
  • 特願2018-044752 (2018.3.12) JP
発明の名称 (英語) AGENT FOR TREATMENT OR PREVENTION OF MUSCULAR-DYSTROPHY-RELATED CARDIOMYOPATHY NEW
発明の概要(英語) An agent for the treatment or prevention of muscular-dystrophy-related cardiomyopathy, the agent containing as an active ingredient a compound represented by formula (1) or a phamacologically acceptable salt or solvate thereof. [In formula (1), R1, R2, R3, R4, and R5 each independently are C1-6 alkyl groups that may contain halogen atoms.]
従来技術、競合技術の概要(英語) BACKGROUND ART
Muscular dystrophy is associated, and reproducing are repeated to necrosis of the muscle fibers, muscle weakness and muscular atrophy gradually to promote the genetic disorder. Wherein, the decrease in the strength than at the time of birth of the admission, congenital muscular dystrophy is called. Congenital muscular dystrophy is, some of the known type, in Japan, congenital muscular dystrophy fukuyama-expression of a high frequency. During the diagnosis of congenital muscular dystrophy fukuyama type, muscle tissue (muscle biopsy) is inspected, congenital muscular dystrophy and other difficult to distinguish from, the existence of abnormality (Fukutin) fukutin gene genetic diagnosis is determined by examining the diagnosis.
Current, congenital muscular dystrophy fukuyama type radical treatment is not on, the ability to maintain even a small amount of motion by the rehabilitation or, joint contractures is mainly prevented (this congratulation) becomes. Also, anticonvulsant medicine to cramps, use of a ventilator mask-nose for disordered breathing, cardiac dysfunction drugs for the treatment, the swallowing with respect to the obstacle such as the stomach and through the use of wax gavages, symptoms of complications with respect to the appropriate treatment is performed, is better than that of the daily life are important.
In this way, a functional failure of the cardiac complications of muscular dystrophy, in particular for cardiomyopathy, treatment is performed in accordance with the condition. However, the treatment, for therapy of coping with the symptoms, is not necessarily a sufficient effect.
Fuku method of abnormal gene, splicing according to the abnormality report is performed (Non-Patent Document 1), which may be treated in the exon trapping method has been suggested. In addition, added by the entity of the sugar chain fukutin become apparent from the (non-patent document 2), a specific sugar chain component and the target is a possibility of the treatment study progresses, the possibility as a therapeutic equivalent to the considered month and labor are required. Any of these, congenital muscular dystrophy fukuyama type of skeletal muscle with respect to the main purpose of the treatment according to the present invention.
  • 出願人(英語)
  • ※2012年7月以前掲載分については米国以外のすべての指定国
  • OKAYAMA UNIVERSITY
  • KOBE UNIVERSITY
  • 発明者(英語)
  • KATANOSAKA, Yuki
  • NARUSE, Keiji
  • UJIHARA, Yoshihiro
  • TODA, Tatsushi
  • KANAGAWA, Motoi
国際特許分類(IPC)
指定国 National States: AE AG AL AM AO AT AU AZ BA BB BG BH BN BR BW BY BZ CA CH CL CN CO CR CU CZ DE DJ DK DM DO DZ EC EE EG ES FI GB GD GE GH GM GT HN HR HU ID IL IN IR IS JO JP KE KG KH KN KP KR KW KZ LA LC LK LR LS LU LY MA MD ME MG MK MN MW MX MY MZ NA NG NI NO NZ OM PA PE PG PH PL PT QA RO RS RU RW SA SC SD SE SG SK SL SM ST SV SY TH TJ TM TN TR TT TZ UA UG US UZ VC VN ZA ZM ZW
ARIPO: BW GH GM KE LR LS MW MZ NA RW SD SL SZ TZ UG ZM ZW
EAPO: AM AZ BY KG KZ RU TJ TM
EPO: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
OAPI: BF BJ CF CG CI CM GA GN GQ GW KM ML MR NE SN ST TD TG

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