Rinsho Shinkeigaku (Clinical Neurology)

Symposium 14

The infrastructure for the clinical research of muscular dystrophies: Remudy and MDCTN

En Kimura, M.D., Ph.D.1), Harumasa Nakamura, M.D.1), Satomi Mitsuhashi, M.D., Ph.D.1), Fumi Takeuchi, M.D.1), Madoka Mori-Yoshimura, M.D., Ph.D.1), Reiko Shimizu, M.D., Ph.D.1)2), Hirofumi Komaki, M.D., Ph.D.1), Yukiko K Hayashi, M.D., Ph.D.3), Ichizo Nishino, M.D., Ph.D.1), Mitsuru Kawai, M.D.4), and Shin'ichi Takeda, M.D., Ph.D.1)

1)National Center of Neurology and Psychiatry, Japan
2)Department of Pediatrics, Tokyo Women's Medical University
3)Department of Neurophysiology, Tokyo Medical University
4)NHO Higashi-Saitama Hospital

Remudy, operated by the NCNP, runs two national registries for Dystrophinopathy and GNE myopathy in Japan under the collaboration with the TREAT-NMD alliance. The aim is to construct the clinical research infrastructure and accelerate the clinical development research for these rare diseases. We successfully provide the data sets for the feasibility studies, send out the appropriate information of the clinical trials for the candidates to speed up the recruitment for trials, collaboration with the Muscular Dystrophy Clinical Trial Network: MDCTN, as well as present the natural history and epidemiological data of the rare diseases with a new 'registry based' research style. Remudy provides a prototype of the clinical research infrastructure to over come the rare and incurable diseases.
Full Text of this Article in Japanese PDF (261K)

(CLINICA NEUROL, 54: 1069|1070, 2014)
key words: Remudy, Muscular dystrophy, International hermonization, rare diseases, TREAT-NMD

(Received: 23-May-14)