Dr. Shin’ichi Takeda is currently the honorary director general at the National Institute of Neuroscience, National Center of Neurology and Psychiatry (NCNP) in Tokyo. He was initially trained as a clinical Neurologist and then received a Ph.D. degree in Muscle Biology in 1981. He had a long time laboratory experience including Pasteur Institute Paris (1987-1992), and focused his research on development of molecular therapy of Duchenne muscular dystrophy (DMD) since he came back to Japan and has gotten the position in NCNP in 1992. He showed a proof of concept study of exon skipping together with Dr. Eric Hoffman’s research team by using dystrophic dogs that he established (Ann Neurol, 2009). Since then, he expanded his experience in preclinical study to clinical medicine. Indeed, he successfully finished the early phase clinical trial of exon 53 skipping of the dystrophin gene among DMD patients in Japan (Sci Transl Med, 2018). Next phase trials were successfully carried both in Japan and in US under the collaboration with Nippon Shinyaku Co. Ltd. / NS-Pharma (Ann Clin Transl Neurol, in press). The exon 53 skipping drug, Viltolarsen, has been finally approved either by PMDA in Japan or by FDA in US very recently. He has been engaged in TREAT-NMD activities in various aspects for a long time since 2007 mainly as an active member, and he was also working as an associate editor for review of J. Neuromuscular Diseases since 2013, and an associate editor of Am. J. Pathology since 2014.