Title:
AGENT FOR TREATMENT OF MYOTONIC DYSTROPHY
Document Type and Number:
WIPO Patent Application WO/2017/010382
Kind Code:
A1
Abstract:
The present invention provides an agent for the treatment of myotonic dystrophy having as an active ingredient at least one compound selected from the group consisting of erythromycin, clarithromycin, and azithromycin, a pharmacologically acceptable salt or hydrate thereof, or a prodrug thereof as an agent for the treatment of myotonic dystrophy that can be administered over an extended period of time and is highly safe and able to improve the symptoms of myotonic dystrophy by suppressing the splicing abnormalities that are the cause of myotonic dystrophy and increasing the normal splicing products.
Inventors:
NAKAMORI MASAYUKI (JP)
MOCHIZUKI HIDEKI (JP)
MOCHIZUKI HIDEKI (JP)
Application Number:
PCT/JP2016/070067
Publication Date:
January 19, 2017
Filing Date:
July 07, 2016
Export Citation:
Assignee:
UNIV OSAKA (JP)
International Classes:
A61K31/7048; A61K31/7052; A61P21/04; A61P43/00
Domestic Patent References:
| WO2014102778A2 | 2014-07-03 |
Foreign References:
| JP2014502598A | 2014-02-03 |
Other References:
RONNBLOM A. ET AL.: "Gastric emptying in myotonic dystrophy", EUROPEAN JOURNAL OF CLINICAL INVESTIGATION, vol. 32, 2002, pages 570 - 574, XP055345505
MASANORI TAKAHASHI ET AL.: "Therapeutic development in myotonic dystrophy", CLINICAL NEUROLOGY, vol. 54, no. 12, 2014, pages 1077 - 1079, XP055345507
NAKAMORI M. ET AL.: "Stabilization of expanded (CTG) . (CAG) repeats by antisense oligonucleotides", MOLECULAR THERAPY, vol. 19, no. 12, 2011, pages 2222 - 2227, XP008153690
COONROD A.L. ET AL.: "Reducing levels of toxic RNA with small molecules", ACS CHEMICAL BIOLOGY, vol. 8, no. 11, 2013, pages 2528 - 2537, XP055345510
NAKAMORI M. ET AL.: "Oral administration of erythromycin decreases RNA toxicity in myotonic dystrophy", A NNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY, vol. 3, no. 1, pages 42 - 54, XP055345513
MASANORI TAKAHASHI ET AL.: "Therapeutic development in myotonic dystrophy", CLINICAL NEUROLOGY, vol. 54, no. 12, 2014, pages 1077 - 1079, XP055345507
NAKAMORI M. ET AL.: "Stabilization of expanded (CTG) . (CAG) repeats by antisense oligonucleotides", MOLECULAR THERAPY, vol. 19, no. 12, 2011, pages 2222 - 2227, XP008153690
COONROD A.L. ET AL.: "Reducing levels of toxic RNA with small molecules", ACS CHEMICAL BIOLOGY, vol. 8, no. 11, 2013, pages 2528 - 2537, XP055345510
NAKAMORI M. ET AL.: "Oral administration of erythromycin decreases RNA toxicity in myotonic dystrophy", A NNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY, vol. 3, no. 1, pages 42 - 54, XP055345513
Attorney, Agent or Firm:
IWATANI, Ryo (JP)
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