臨床神経学

<シンポジウム04―1>神経内科領域における前方向的コホート研究から見えてきたもの

筋ジストロフィーレジストリー(Remudy);
患者の自発的登録システム構築と海外との連携

中村 治雅1), 川井 充2)

1)独立行政法人国立精神・神経医療研究センター病院神経内科
2)独立行政法人国立病院機構東埼玉病院神経内科〔〒187―8551 東京都小平市小川東町4―1―1〕

The development of orphan medicines presents many challenges. Clinical trials with new therapeutic strategies are now being planned and conducted for many orphan diseases such as Duchenne and Becker muscular dystrophy (DMD/BMD). However, since adequate numbers of patients are needed to achieve significant results for clinical trials, patient registries are an important infrastructure worldwide, especially in the case of rare diseases. We developed a national registry of Japanese DMD/BMD patients in collaboration with European research network, TREAT-NMD. Japanese registry adopts a self-report system and this database includes clinical data and molecular genetic data. The main purpose of this registry is the effective recruitment of eligible patients for a clinical trial and may provide timely information to individual patients. The registry data gives us more detailed knowledge of natural history, epidemiology, and clinical care. This registry will gain trial readiness in Japan and accelerate more effectively harmonization with other countries to fight orphan diseases.
Full Text of this Article in Japanese PDF (201K)

(臨床神経, 51:901−902, 2011)
key words:筋ジストロフィー,患者登録,レムデイー(Remudy),トリートエヌエムデイー(TREAT-NMD),希少疾病用医薬品

(受付日:2011年5月19日)