一次情報の記録
決定級トップライン結果
uniQure、Huntington病におけるAMT-130の第I/II相36ヶ月データを報告
36ヶ月トップライン解析では、高用量AMT-130が事前に規定された主要評価項目（cUHDRS）および主要副次評価項目（TFC）において、傾向スコアマッチング外部対照群と比較して達成し、疾患進行の統計学的に有意な抑制およびベースラインを下回るCSF NfLレベルを示した。これらのデータは、FDAとの加速承認経路に関する継続的な協議を支持し、AMT-130をHDに対する初の疾患修飾療法候補として位置づける。
主要事実
出典の抜粋
EX-99.1 2 qure-20260113xex99d1.htm EX-99.1 Sophia – Huntington’s Disease Community Advocate Leadership in Gene Therapy January 2026 Exhibit 99.1 LEADERSHIP IN GENE THERAPY JANUARY 2026 | 2 Disclaimer This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect，” “goal,” “intend,” “look forward to,” “may,” “plan,” “potential,” “predict,” “project,” “should,” "will,” “would” and similar expressions and the negatives of those terms. Forward-looking st