NEJM· Marianna Fontana Ahmad Masri Scott D. Solomon Francesco Cappelli Margot K. Davis Pablo García-Pavía Julian D. Gillmore Mazen Hanna Laura Obici Brett W. Sperry Nobuhiro Tahara Márcia Waddington-Cruz Olga Azevedo Thibaud Damy Edileide de Barros Correia Michel G. Khouri Andriana P. Nikolova Stefan Störk Tom Conrad Qingqing Yang Brian L. Claggett Jersey Chen Gary T. Brice Sotirios Tsimikas and Mathew S. Maurerthe CARDIO-TTRansform Investigators*·· 2026-08-28评分0
Eplontersen 用于转甲状腺素蛋白淀粉样变性心肌病
Eplontersen for Transthyretin Amyloid Cardiomyopathy
导读
Eplontersen 在转甲状腺素蛋白淀粉样变性心肌病患者中显示出显著疗效。该研究达到了主要终点，与安慰剂相比显著改善了患者的综合功能评分。安全性特征与已知数据一致。
来源：NEJM · nejm.org