eplontersen是一种配体偶联反义(LICA)药物，可减少转甲状腺素蛋白（TTR）的产生。 ATTR淀粉样变性（图片来源：clevelandclinic.org ...

Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on CRISPR-Cas9 (clustered regularly ...

转甲状腺素蛋白淀粉样变性（ATTR）表型多样，测量其健康结局存在挑战。研究人员评估 ATTR-QOL 问卷影响量表的效度和信度，发现其可靠有效，还更新了问卷。这有助于临床和研究中更好地评估患者生活质量。 在医学领域，转甲状腺素蛋白淀粉样变性（ATTR）是一 ...

Cardiac transthyretin amyloidosis (also called ATTR amyloidosis cardiomyopathy or ATTR-CM) is a progressive disease and a cause of fatal heart failure resulting from extracellular myocardial ...

ATTR phenotypes vary by genotype as well as by gene penetrance, age, and geographic location. A new analysis examined data from untreated, symptomatic patients who had 1 of 4 variants: F64L, I68L, ...

BridgeBio (BBIO) Pharma announced that the U.S. Food and Drug Administration approved Attruby, an orally-administered near-complete stabilizer of Transthyretin, or TTR, for the treatment of adults ...