SAFETY OF INOTERSEN TREATMENT IN PATIENTS WITH TRANSTHYRETIN AMYLOID CARDIOMYOPATHY
JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY（2019）
摘要
Transthyretin amyloidosis (ATTR) is a protein misfolding disorder that causes cardiomyopathy (CM) and results in significant morbidity and mortality. Inotersen is an antisense oligonucleotide inhibitor of transthyretin protein production.NEURO-TTR ([NCT01737398][1]) was a randomized, controlled
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