title: [Infrastructure for the clinical research of muscular dystrophies: remudy and MDCTN].
abstract: 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.
authors.affiliation: National Center of Neurology and Psychiatry, Japan.
authors.forename: En
authors.initials: E
authors.lastname: Kimura
authors.forename: Harumasa
authors.initials: H
authors.lastname: Nakamura
authors.forename: Satomi
authors.initials: S
authors.lastname: Mitsuhashi
authors.forename: Fumi
authors.initials: F
authors.lastname: Takeuchi
authors.forename: Madoka
authors.initials: M
authors.lastname: Mori-Yoshimura
authors.forename: Reiko
authors.initials: R
authors.lastname: Shimizu
authors.forename: Hirofumi
authors.initials: H
authors.lastname: Komaki
authors.forename: Yukiko K
authors.initials: YK
authors.lastname: Hayashi
authors.forename: Ichizo
authors.initials: I
authors.lastname: Nishino
authors.forename: Mitsuru
authors.initials: M
authors.lastname: Kawai
authors.forename: Shin'ichi
authors.initials: S
authors.lastname: Takeda
mesh_terms: D002986:Clinical Trials as Topic; D007391:International Cooperation; D007564:Japan; D009136:Muscular Dystrophies / Q000453:epidemiology; D012042:Registries; D012106:Research