NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy

Author:

Watanabe Naoki,Nagata Tetsuya,Satou Youhei,Masuda Satoru,Saito Takashi,Kitagawa Hidetoshi,Komaki Hirofumi,Takagaki Kazuchika,Takeda Shin’ichi

Funder

Grants-in-Aid for Research on Nervous and Mental Disorders

Health and Labour Sciences Research Grants for Translation Research

Comprehensive Research on Disability Health and Welfare

Ministry of Health, Labour and Welfare of Japan

Grants for Promoting Clinical Trials for the Development of New Drugs and Medical Devices

Health and Labour Science Research Grants for Comprehensive Research on Persons with Disabilities

Japan Agency for Medical Research and Development

Takeda Science Foundation

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference25 articles.

1. A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy;Mah;Neuromuscul. Disord.,2014

2. The muscular dystrophies;Emery;Lancet,2002

3. Therapeutic advances in muscular dystrophy;Leung;Ann. Neurol.,2013

4. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy;Mendell;Ann. Neurol.,2016

5. Sarepta Therapeutics (2015). Sarepta Therapeutics announces FDA has filed eteplirsen NDA for the potential treatment of Duchenne muscular dystrophy for patients amenable to exon 51 skipping. http://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-fda-has-filed-eteplirsen-nda.

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