Results 111 to 120 of about 2,075,366 (204)

A phase 3 clinical trial with ISIS-TTRRx, a 2nd-generation antisense oligonucleotide targeting transthyretin (TTR), for the treatment of TTR amyloid cardiomyopathy [PDF]

, 2015

core   +1 more source

Natural history, Staging and Prognosis in Systemic Amyloidosis [PDF]

Background: Systemic amyloidosis is a rare disorder of protein misfolding and aggregation ranging from indolent localised disease to rapidly progressive multi-organ failure. Systemic light-chain (AL) and transthyretin (ATTR) amyloidosis are the commonest
Law, Steven John
core  

Concomitant Cardiac Transthyretin Amyloidosis and Coronary Artery Disease---Imaging Features and Pitfalls

CJC Open
The coexistence of coronary artery disease (CAD) and transthyretin cardiac amyloidosis (ATTR-CM) is increasingly recognized in clinical practice, especially in older adults.
Nicolas Lavoie, MD, Omar Idrissi, MD, Julien Ternacle, MD, PhD, Kim O’Connor, MD, Andreanne Powers, MD, Alexis Jacquier, MD, PhD, Francis Morin, MD, Michel Tessier, MD, Tomas Cieza, MD, Jean-Michel Paradis, MD, Élisabeth Bedard, MD, Mohamed Marzouk, MD, Jonathan Beaudoin, MD, Nancy Coté, PhD, Mathieu Bernier, MD, Philippe Pibarot, DVM, PhD, Marie-Annick Clavel, DVM, PhD, Erwan Salaun, MD, PhD   +17 more
doaj   +1 more source

A series of cases of transthyretin amyloid cardiomyopathy with negative bone scintigraphy but a confirmed positive endomyocardial biopsy

Orphanet Journal of Rare Diseases
Background Bone scintigraphy (BS) is established as an accurate, non-invasive method for the diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM). In a real-life setting, however, some patients with no cardiac uptake on BS turn out to have cardiac-
Antoine Fraix, Emmanuel Itti, Amira Zaroui, Mounira Kharoubi, Elsa Poullot, Lionel Lerman, Soulef Guendouz, Olivier Huttin, Thibaud Damy, Arnault Galat   +9 more
doaj   +1 more source

CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy [PDF]

BACKGROUND: 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 interspaced short palindromic repeats and ...
Adams, David, Fontana, Marianna, Gane, Edward J, Gillmore, Julian D, Gutstein, David E, Haagensen, Alexandra, Kachadourian, Jessica, Lebwohl, David, Leung, Adia, Olbertz, Joy, Pilebro, Björn, Razvi, Yousuf, Rocha, Ricardo, Smith, Derek, Solomon, Scott D, Sonderfan, Alison, Täubel, Jörg, Walsh, Liron, Xu, Yuanxin, Zhu, Peijuan   +19 more
core  

The potency of AG10 in stabilizing transthyretin is driven by interactions mimicking the disease-suppressing T119M variant [PDF]

, 2019
Transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM) is a fatal disease with no available disease-modifying therapies. While destabilizing TTR mutations increase the risk of developing ATTR-CM, the naturally occurring Thr119Met (T119M) variant stabilizes
Liang, Dengpan
core   +1 more source

Artificial intelligence-based echocardiographic assessment for monitoring disease progression in transthyretin cardiac amyloidosis [PDF]

Aims: In transthyretin amyloid cardiomyopathy (ATTR-CM), reduced stroke volume (SV) portends a poor prognosis. Artificial intelligence (AI) enables rapid, standardized assessment of left ventricular outflow tract velocity-time integral (LVOT-VTI), which ...
Aimo, Alberto, Bandera, Francesco, Emdin, Michele, Ezekowitz, Justin, Fontana, Marianna, Frost, Matthew, Gillmore, Julian D, Hawkins, Philip N, Ioannou, Adam, Iyer, Surabhi Bhaskar, Lachmann, Helen, Lam, Carolyn SP, Lim, Sze Chi, Mansell, Josephine, Martinez-Naharro, Ana, Moody, William, Porcari, Aldostefano, Razvi, Yousuf, Sezer, Irem, Sheikh, Awais, Solomon, Scott David, Venneri, Lucia, Wechelakar, Ashutosh, Whelan, Carol   +23 more
core  

<i>In-vivo</i> CRISPR editing for ATTR-CM: therapeutic transthyretin silencing through hepatic genome correction. [PDF]

Ann Med Surg (Lond)
Tahir A, Imtiaz E, Ali FZ, Khan MS, Mahato RK.   +4 more
europepmc   +1 more source

Tc-99 m Pyrophosphate Uptake in Atrial Amyloidosis With Hemorrhagic Mass　- A Diagnostic Pitfall Mimicking ATTR-CM. [PDF]

Circ Rep
Fujimoto N, Moriwaki K, Makino M, Dohi K.   +3 more
europepmc   +1 more source

Comparison of in-clinic assessment of 6MWT by conventional method and using wearable sensors for patients with ATTR-CM. [PDF]

Future Cardiol
Soman P, Khouri MG, Lenihan D, Reyentovich A, Sperry BW, Sowalsky K, Bai Y, Du J, Katz L, Siddhanti S, Fox JC.   +10 more
europepmc   +1 more source