Results 61 to 70 of about 231,717 (193)

Impact of Baseline Polyneuropathy Severity on Eplontersen Efficacy in the NEURO‐TTRansform Clinical Trial

Journal of the Peripheral Nervous System, Volume 31, Issue 3, September 2026.
ABSTRACT Background and Aims In the NEURO‐TTRansform clinical trial (NCT04136184), eplontersen improved neuropathy impairment and quality of life (QoL) through Week 66 versus the NEURO‐TTR historical placebo in patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv‐PN).
Thomas H. Brannagan III, Markus Weiler, Márcia Waddington Cruz, Sami Khella, John L. Berk, Morie Gertz, Ahmad Masri, Mathew S. Maurer, Jonas Wixner, Jonatan Nåtman, T. Jesse Kwoh, Maksym Pola, Laura Obici   +12 more
wiley   +1 more source

Amyloid polyneuropathy caused by wild‐type transthyretin [PDF]

, 2015
IntroductionAmyloidosis derived from transthyretin (TTR) molecules is typically caused by mutations of the TTR gene.MethodsWe describe an elderly patient with a severe length-dependent polyneuropathy that unexpectedly proved to be caused by wild-type ...
Lam, Lynda, Margeta, Marta, Layzer, Robert   +2 more
core   +1 more source

Prevalence, characteristics, and mortality of patients with transthyretin amyloid cardiomyopathy in the Nordic countries

ESC Heart Failure, 2022
Aims Transthyretin amyloid cardiomyopathy (ATTR‐CM) is a progressive condition caused by deposition of transthyretin amyloid fibrils in the heart and is associated with poor quality of life and a shortened lifespan.
Rosa Lauppe, Johan Liseth Hansen, Anna Fornwall, Katarina Johansson, Mark H. Rozenbaum, Anne Mette Strand, Merja Väkeväinen, Johanna Kuusisto, Einar Gude, J. Gustav Smith, Finn Gustafsson   +10 more
doaj   +1 more source

Efficacy and Limitations of Corneal Confocal Microscopy in Evaluating ATTRv‐PN

Brain and Behavior, Volume 16, Issue 8, August 2026.
ABSTRACT Objective Driven by pathogenic variants in the TTR gene, hereditary transthyretin amyloidosis with polyneuropathy (ATTRv‐PN) presents as a systemic autosomal dominant disorder. This cross‐sectional study aimed to investigate the clinical utility of in vivo corneal confocal microscopy (CCM) for assessing structural changes in corneal nerve ...
Yize Li, Kang Du, Wenjing Song, Difang Shi, Xujun Chu, Ping Li, Zhenyu Li, He Lv, Wei Zhang, Zhaoxia Wang, Yun Yuan, Yuan Wu, Lingchao Meng   +12 more
wiley   +1 more source

Cerebrovascular Involvement in Transthyretin Amyloid Cardiomyopathy

Journal of Clinical Medicine
Background: Intracardiac thrombosis is common in transthyretin amyloid cardiomyopathy (ATTR-CM), and patients are at risk for thromboembolic events. However, silent cerebral infarcts and the extent of cerebral small vessel disease in patients with cardiac amyloidosis are unknown.
Lukas Haider, Lore Schrutka, Emanuele Tommasino, Nicolas Avanzini, Sven Hauck, Nikolaus Nowak, Christian Hengstenberg, Diana Bonderman, Majda Thurnher   +8 more
openaire   +2 more sources

3 Discordant Extracardiac Amyloid Proteins Highlighting Bystander Deposition in a Case of Light-Chain Amyloid Cardiomyopathy

JACC: Case Reports
Background: Accurate differentiation of immunoglobulin light-chain (AL) from transthyretin (ATTR) amyloid cardiomyopathy is critical, as misclassification may delay appropriate therapy and worsen outcomes.
Roxanne Coderre, MD, Gregorio Tersalvi, MD, Rosalyn O. Adigun, MD, Barry A. Boilson, MD, Daniel D. Borgeson, MD, Alfredo L. Clavell, MD, Ian C. Chang, MD, Nadine H. Abdallah, MD, Martha Grogan, MD, Omar F. AbouEzzeddine, MDCM, MS   +9 more
doaj   +1 more source

Catheter Ablation for Atrial Fibrillation in Cardiac Amyloidosis: A Systematic Review and Meta‐Analysis

Journal of Arrhythmia, Volume 42, Issue 4, August 2026.
This systematic review and meta‐analysis of 13 observational studies found that catheter ablation was associated with lower odds of all‐cause mortality compared with medical management in cardiac amyloidosis and atrial fibrillation. Freedom from atrial arrhythmia after ablation was approximately 50%, with no significant associations for heart failure ...
Adivitch Sripusanapan, Natee Deepan, Bheem Pongsukvechkul, Suphatcha Kittiwongsopon, Min Choon Tan, Pattara Rattanawong, Dan Sorajja, Win‐Kuang Shen, Ronpichai Chokesuwattanaskul, Narut Prasitlumkum   +9 more
wiley   +1 more source

Structure-Based Probe Reveals the Presence of Large Transthyretin Aggregates in Plasma of ATTR Amyloidosis Patients

JACC: Basic to Translational Science
Summary: Amyloidogenic transthyretin (ATTR) amyloidosis is a relentlessly progressive disease caused by the misfolding and systemic accumulation of amyloidogenic transthyretin into amyloid fibrils.
Rose Pedretti, BS, Lanie Wang, BS, Anna Yakubovska, MS, Qiongfang S. Zhang, BS, Binh Nguyen, PhD, Justin L. Grodin, MD, Ahmad Masri, MD, Lorena Saelices, PhD   +7 more
doaj   +1 more source

Multi‐Omics‐Guided Design and Safety Engineering of Nucleic Acid Therapeutics: From Molecular Perturbation to Predictive Toxicology and Precision Translation

Chemical Biology &Drug Design, Volume 108, Issue 2, August 2026.
Multi‐omics (proteomics, metabolomics, and single‐cell/spatial transcriptomics) provides a systems framework to distinguish on‐target efficacy from off‐target and toxicity signatures of chemically modified nucleic acid therapeutics, enabling omics‐guided structure–activity and safety optimisation from discovery to clinical translation. ABSTRACT Nucleic
Gurjit Kaur Bhatti, Anushka Verma, Komal Devi, Naina Khullar, Inderpal Singh Sidhu, Umesh Chand Singh Yadav, Jasvinder Singh Bhatti   +6 more
wiley   +1 more source

Science in the News - AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V1221 transthyretin [PDF]

, 2018
Penchala, Sravan C., et al. “AG10 Inhibits Amyloidogenesis and Cellular Toxicity of the Familial Amyloid Cardiomyopathy-Associated V122I Transthyretin.” PNAS, National Academy of Sciences, 11 June 2013, www.pnas.org/content/110/24 ...
Saelee, Tiger
core