BRAIN and NERVE－神経研究の進歩69巻8号

2017年08月発行

特集 遺伝性脊髄小脳失調症の病態と治療展望

薬物療法の現状と最近の試み—フリードライヒ運動失調症とマシャド・ジョセフ病を中心に

著者： 矢部一郎¹ 佐々木秀直¹

所属機関： ¹北海道大学大学院医学研究院神経病態学分野神経内科

ページ範囲：P.913 - P.924

文献概要

脊髄小脳変性症の根本的治療法は見出されておらず，小脳性運動失調に対する対症療法として，酒石酸プロチレリンとタルチレリン水和物が保険適用として認可されているのみである。近年病態解明が進み，その成果を踏まえて複数の臨床試験が実施されている。その有効性を評価するためのバイオマーカーの開発も進んでいる。本論では，フリードライヒ運動失調症およびマシャド・ジョセフ病に対する最近の薬物療法の試みについて紹介する。

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