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

Foreign code F190009982
File No. (S2018-0451-N0)
Posted date Oct 28, 2019
Country WIPO
International application number 2019JP009742
International publication number WO 2019176864
Date of international filing Mar 11, 2019
Date of international publication Sep 19, 2019
Priority data
  • P2018-044752 (Mar 12, 2018) JP
Title AGENT FOR TREATMENT OR PREVENTION OF MUSCULAR-DYSTROPHY-RELATED CARDIOMYOPATHY NEW_EN
Abstract 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.]
Outline of related art and contending technology 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.
Scope of claims (In Japanese)[請求項1]
 式(1)で示される化合物又はその薬学的に許容される塩若しくは溶媒和物を有効成分として含有する、筋ジストロフィー関連心筋症の治療又は予防剤。
(省略)
[式(1)中、R 1、R 2、R 3、R 4及びR 5は、それぞれ独立に、ハロゲン原子を含んでいてもよい炭素数1~6のアルキル基である。]

[請求項2]
 式(1)で示される化合物が、式(2)で示されるコルヒチンである請求項1に記載の治療又は予防剤。

[請求項3]
 前記筋ジストロフィーが、ジストログリカン異常症である、請求項1又は2に記載の治療又は予防剤。

[請求項4]
 前記ジストログリカン異常症が、福山型先天性筋ジストロフィー、ウォーカー・ワールブルグ(Walker-Warburg)症候群、筋眼脳病又は肢帯型筋ジストロフィーである、請求項3に記載の治療又は予防剤。
  • Applicant
  • ※All designated countries except for US in the data before July 2012
  • OKAYAMA UNIVERSITY
  • KOBE UNIVERSITY
  • Inventor
  • KATANOSAKA, Yuki
  • NARUSE, Keiji
  • UJIHARA, Yoshihiro
  • TODA, Tatsushi
  • KANAGAWA, Motoi
IPC(International Patent Classification)
Specified countries 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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