Affiliation: National Institute of Neuroscience
Shimizu Motohashi Y, Komaki H, Motohashi N, Takeda S, Yokota T, Aoki Y. Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches. J Pers Med. 2019;9: pubmed publisher
..Here we review the current status of therapeutic approaches for DMD, focusing on therapeutic approaches that can restore dystrophin. ..
Sugie K, Komaki H, Eura N, Shiota T, Onoue K, Tsukaguchi H, et al
. A Nationwide Survey on Danon Disease in Japan. Int J Mol Sci. 2018;19: pubmed publisher
..Cardiomyopathy is the most significant prognostic factor and the main cause of death. Our findings suggest that the present survey can extend our understanding of the clinical features of this rare disease. ..
Takeuchi F, Komaki H, Nakamura H, Yonemoto N, Kashiwabara K, Kimura E, et al
. Trends in steroid therapy for Duchenne muscular dystrophy in Japan. Muscle Nerve. 2016;54:673-80 pubmed publisher
..This study revealed a transition over time in steroid use from expert opinion to evidence-based recommendation. Clinical research should be encouraged to optimize medication worldwide. Muscle Nerve 54: 673-680, 2016. ..
Motoki T, Shimizu Motohashi Y, Komaki H, Mori Yoshimura M, Oya Y, Takeshita E, et al
. Treatable renal failure found in non-ambulatory Duchenne muscular dystrophy patients. Neuromuscul Disord. 2015;25:754-7 pubmed publisher
..The findings suggest that non-ambulatory DMD patients are at a risk of reduced kidney perfusion, which potentially leads to prerenal failure. Therefore, in DMD patients, dehydration signs and CysC levels should be monitored. ..
Goto M, Komaki H, Takeshita E, Abe Y, Ishiyama A, Sugai K, et al
. Long-term outcomes of steroid therapy for Duchenne muscular dystrophy in Japan. Brain Dev. 2016;38:785-91 pubmed publisher
..This is the first assessment of long-term outcomes of different steroid therapy regimens in Japanese DMD patients. Benefits and side effects, except height, did not differ significantly between steroid regimens. ..
Shimizu R, Ogata K, Tamaura A, Kimura E, Ohata M, Takeshita E, et al
. Clinical trial network for the promotion of clinical research for rare diseases in Japan: muscular dystrophy clinical trial network. BMC Health Serv Res. 2016;16:241 pubmed publisher
..Thus, our network will be able to contribute with international research activity, which can lead to an improvement of neuromuscular disease treatment in Japan. ..