title: [Remudy].
abstract: Remudy (Registry of Muscular Dystrophy), operated by NCNP (the National Center of Neurology and Psychiatry), Japan, runs two national registries for Dystrophinopathy and GNE myopathy in collaboration with the TREAT-NMD (Translational Research in Europe--Assessment and Treatment of Neuromuscular Disease) alliance. The aim of Remudy is to construct a clinical research infrastructure that accelerates the pace of clinical development research for these rare diseases. We successfully provide data sets for feasibility studies, send out appropriate information about clinical trials to speed up the recruitment of candidates, as well as present the natural history and epidemiology data of these rare diseases using a new 'registry based' research style. Remudy presents a prototype model of the clinical research infrastructure to overcome these rare and incurable diseases.
authors.affiliation: Translational Medical Center, National Center of Neurology and Psychiatry.
authors.forename: En
authors.initials: E
authors.lastname: Kimura
authors.forename: Harumasa
authors.initials: H
authors.lastname: Nakamura
authors.forename: Ichizo
authors.initials: I
authors.lastname: Nishino
mesh_terms: D035843:Biomedical Research / Q000706:statistics & numerical data; D003299:Cooperative Behavior; D005060:Europe; D006801:Humans; D007564:Japan; D009136:Muscular Dystrophies; D012042:Registries / Q000706:statistics & numerical data; D057170:Translational Research, Biomedical / Q000706:statistics & numerical data