BRAIN and NERVE－神経研究の進歩72巻1号

2020年01月発行

特集 神経難病をクスリで治す—薬物開発の現況と近未来への展望

筋萎縮性側索硬化症における創薬開発の現状と展望

著者： 漆谷真¹

所属機関： ¹滋賀医科大学内科学講座脳神経内科

ページ範囲：P.13 - P.22

文献概要

筋萎縮性側索硬化症（ALS）は脳神経内科における最難治性疾患である。しかし家族性ALSの原因遺伝子の同定，モデル動物やiPS細胞を用いた病態解析が進展し新たな治療候補薬が次々と見出され，治験が実現している。多くは検証的段階であるが，家族性ALSに対する核酸治療など原因に根ざした有望な治療も出現間近である。わが国発のエダラボンが抗ALS薬として海外で採用され始め，いまやALSは最も治療研究開発が盛んな疾患の1つである。

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