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アイオニス・ファーマシューティカルズ社、アレキサンダー病の臨床試験でジルガネルセンの重要な結果を発表

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-- イオニス・ファーマシューティカルズ(IONS)は、希少でしばしば致死的な神経変性疾患であるアレキサンダー病を患う5歳以上の患者を対象とした臨床試験で、同社のジルガネルセン50mgが統計的に有意な効果を示したと発表した。 同社は火曜日の声明で、この重要な臨床試験が主要評価項目を達成し、10メートル歩行テストで評価した歩行速度の臨床的に意義のある安定化が認められたと述べた。 同社によると、粗大運動機能測定88(GMFM-88)の新たなデータでは、ジルガネルセンを投与された2歳から4歳の幼児は、対照群の幼児よりも粗大運動機能が改善したことが示された。 イオニスは、ジルガネルセンは良好な安全性と忍容性プロファイルを示し、ほとんどの有害事象は軽度または中等度であったと述べた。 イオニスによると、この薬剤は米国食品医薬品局(FDA)で優先審査を受けており、9月22日までに承認の決定が下される見込みである。

Price: $75.13, Change: $+0.25, Percent Change: +0.34%

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