作者
Julian D. Gillmore,Daniel P. Judge,Francesco Cappelli,Marianna Fontana,Pablo García‐Pavía,Simon Gibbs,Martha Grogan,Mazen Hanna,James E. Hoffman,Ahmad Masri,Mathew S. Maurer,Jose Nativi‐Nicolau,Laura Obici,Steen Hvitfeldt Poulsen,Frank W. Rockhold,Keyur B. Shah,Prem Soman,Jyotsna Garg,Karen Chiswell,Haolin Xu,Xiaofan Cao,Theodore C. Lystig,Uma Sinha,Jonathan C. Fox
摘要
Transthyretin amyloid cardiomyopathy is characterized by the deposition of misfolded monomeric transthyretin (TTR) in the heart. Acoramidis is a high-affinity TTR stabilizer that acts to inhibit dissociation of tetrameric TTR and leads to more than 90% stabilization across the dosing interval as measured ex vivo.
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