Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study-Reference-Cited by-同舟云学术

Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study

Published:2020-12 Issue:12 Volume:7 Page:2393-2408
ISSN:2328-9503
Container-title:Annals of Clinical and Translational Neurology
language:en
Short-container-title:Ann. Clin. Transl. Neurol.

Author:

Komaki Hirofumi¹^ORCID,Takeshima Yasuhiro²,Matsumura Tsuyoshi³,Ozasa Shiro⁴,Funato Michinori⁵,Takeshita Eri⁶,Iwata Yasuyuki⁷,Yajima Hiroyuki⁷,Egawa Yoichi⁸,Toramoto Takuya⁸,Tajima Masaya⁸,Takeda Shinichi⁹

Affiliation:

1. Translational Medical CenterNational Center of Neurology and Psychiatry Kodaira Tokyo Japan

2. Department of Pediatrics Hyogo College of Medicine Nishinomiya Hyogo Japan

3. Department of Neurology National Hospital Organization Osaka Toneyama Medical Center Toyonaka Osaka Japan

4. Department of Pediatrics Kumamoto University Hospital Kumamoto Kumamoto Japan

5. Department of Pediatrics National Hospital Organization Nagara Medical Center Gifu Japan

6. Department of Child Neurology National Center of Neurology and Psychiatry National Center Hospital Kodaira Tokyo Japan

7. Department of Rehabilitation National Center of Neurology and Psychiatry National Center Hospital Kodaira Tokyo Japan

8. Global Clinical Development Department Nippon Shinyaku Co., Ltd. Kyoto Kyoto Japan

9. National Center of Neurology and Psychiatry Kodaira Tokyo Japan

Publisher

Wiley

Subject

Neurology (clinical),General Neuroscience

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/acn3.51235

Reference27 articles.

1. Advances in genetic therapeutic strategies for Duchenne muscular dystrophy

2. Dystrophin: The protein product of the duchenne muscular dystrophy locus

3. Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals

4. Specific Interaction of the Actin-binding Domain of Dystrophin with Intermediate Filaments Containing Keratin 19

5. The Dystrophin Complex: Structure, Function, and Implications for Therapy

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