Response to therapy with tafamidis 61 mg in patients with cardiac transthyretin amyloidosis: real-world experience since approval [0.03%]
61毫克tafamidis治疗心脏转甲状腺素蛋白淀粉样变性患者的疗效：自获批以来的现实世界经验
Fabian Aus dem Siepen,Christopher Meissner,Eva Hofmann et al.
Fabian Aus dem Siepen et al.
Aims: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive disease that causes heart failure due to amyloid fibril deposition. Tafamidis was approved as the first causal treatment in 2020. We here report on rea...
Glaucoma is not seen at a higher prevalence in age-related transthyretin amyloidosis after race stratification [0.03%]
按种族分层后可见年龄相关转甲状腺素蛋白淀粉样变性患者青光眼的患病率并不高
Noel Estrada-Merly,Mathew S Maurer,Anita D&#x;Souza
Noel Estrada-Merly
Delayed identification of monoclonal protein is associated with early death in isolated cardiac AL amyloidosis [0.03%]
孤立性心脏AL淀粉样变性中单克隆蛋白延迟识别与早期死亡相关
Paolo Milani,Francesca Fabris,Roberta Mussinelli et al.
Paolo Milani et al.
Background: Early identification of immunoglobulin light-chain amyloidosis (AL) is crucial due to its rapid progression. Monoclonal light-chain (M-LC) testing is the first step in the diagnostic workup for patients with s...
Internalisation of immunoglobulin light chains by cardiomyocytes in AL amyloidosis: what can biopsies tell us? [0.03%]
免疫球蛋白轻链的心肌细胞内化在AL型淀粉样变性中起到了什么作用？活检能告诉我们什么？
Mélanie Bézard,Amira Zaroui,Mounira Kharoubi et al.
Mélanie Bézard et al.
Background: Cardiac involvement in systemic light chain amyloidosis (AL) leads to chronic heart failure and is a major prognosis factor. Severe cellular defects are provoked in cardiac cells by tissue-deposited amyloid fi...
Outcomes of venetoclax-based therapy in patients with t(11;14) light chain amyloidosis after failure of daratumumab-based therapy [0.03%]
达雷木单抗治疗失败的t（11;14）轻链淀粉样变患者接受维奈克拉为基础治疗的效果分析
Danai Dima,Michael Hughes,Mark Orland et al.
Danai Dima et al.
Background: Daratumumab's incorporation in the upfront treatment of light chain (AL) amyloidosis has led to daratumumab (dara) refractoriness early in disease course. Patients who experience relapse or have suboptimal res...
Transthyretin monomers: a new plasma biomarker for pre-symptomatic transthyretin-related amyloidosis [0.03%]
稳定不变的血清前神經病變期 ATTR 淀粉樣變異質性標志蛋白ometry单体：ATTR淀粉样变前期无症状阶段的新血浆生物标志物
Diogo Costa-Rodrigues,José P Leite,Maria João Saraiva et al.
Diogo Costa-Rodrigues et al.
Background: Genotyping and amyloid fibril detection in tissues are generally considered the diagnostic gold standard in transthyretin-related amyloidosis. Patients carry less stable TTR homotetramers prone to dissociation...
Identification of isoaspartate-modified transthyretin as potential target for selective immunotherapy of transthyretin amyloidosis [0.03%]
识别出转甲状腺素蛋白异天冬氨酸修饰形式作为选择性免疫治疗转甲状腺素蛋白淀粉样变性的潜在靶点
Janett Köppen,Martin Kleinschmidt,Markus Morawski et al.
Janett Köppen et al.
Background: Numerous studies suggest a progressive accumulation of post-translationally modified peptides within amyloid fibrils, including isoaspartate (isoD) modifications. Here, we generated and characterised novel mon...
Genetic counselling for at-risk family members with hereditary transthyretin amyloidosis: data from a single-centre study [0.03%]
遗传咨询在有遗传性转甲状腺素蛋白淀粉样变病风险的家庭成员中的应用：单一中心研究报告的数据
Katsuya Nakamura,Tsuneaki Yoshinaga,Akiko Sakyu et al.
Katsuya Nakamura et al.
Background: Hereditary transthyretin-related amyloidosis is an autosomal dominant disorder. Recently, disease-modifying therapies (DMTs) have been developed. For at-risk individuals, genetic analysis aids in the early adm...
Stefan Schönland,Per Westermark
Stefan Schönland
Gastrointestinal Amyloid Screening Study (GASS): is screening for amyloid in the gastrointestinal tract useful? [0.03%]
胃肠道淀粉样变筛查研究(GASS)：对胃肠道进行淀粉样变筛查有用吗？
Rola Khedraki,Joseph El-Roumi,Daniela Allende et al.
Rola Khedraki et al.