Kimura En, Nakamura Harumasa, Mitsuhashi Satomi, Takeuchi Fumi, Mori-Yoshimura Madoka, Shimizu Reiko, Komaki Hirofumi, Hayashi Yukiko K, Nishino Ichizo, Kawai Mitsuru, Takeda Shin'ichi
National Center of Neurology and Psychiatry, Japan.
Rinsho Shinkeigaku. 2014;54(12):1069-70. doi: 10.5692/clinicalneurol.54.1069.
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.
由国立神经精神中心运营的Remudy，在与TREAT-NMD联盟的合作下，在日本运营了两个关于肌营养不良症和GNE肌病的全国性登记系统。其目的是构建临床研究基础设施，并加速针对这些罕见疾病的临床开发研究。我们成功为可行性研究提供了数据集，向候选人发送临床试验的适当信息以加快试验招募，与肌肉萎缩症临床试验网络（MDCTN）合作，并以一种新的“基于登记系统”的研究方式呈现罕见疾病的自然史和流行病学数据。Remudy提供了一个临床研究基础设施的原型，以攻克这些罕见且无法治愈的疾病。
