Results 51 to 60 of about 5,684 (155)

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

Tafamidis for transthyretin familial amyloid polyneuropathy : A randomized, controlled trial

, 2012
Objectives: To evaluate the efficacy and safety of 18 months of tafamidis treatment in patients with early-stage V30M transthyretin familial amyloid polyneuropathy (TTR-FAP). Methods: In this randomized, double-blind trial, patients received tafamidis 20
Wilson, Amy, Coelho, Teresa, da Silva, Ana Martins, Maia, Luis F., Grogan, Donna R., Suhr, Ole B.,, Cruz, Marcia Waddington, Lozeron, Pierre, Packman, Jeff, Trigo, Pedro, Chan, Jason, Plante-Bordeneuve, Violaine, Labaudinie, Richard, Conceicao, Isabel Maria, Kelly, Jeffery W., Campistol, Josep M., Schmidt, Hartmut H. -J.   +16 more
core   +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

Tafamidis (Vyndaqel): A Light for FAP Patients

, 2013
Slowing FAP progression: Tafamidis meglumine is a small molecule capable of stabilizing the transthyretin (TTR) tetramer. Tafamidis acts in a similar way to the natural hormone T4, prevents TTR amyloid fibril formation, and offers a potential alternative
Elisabetta Orlandini, Susanna Nencetti, NENCETTI, SUSANNA, ROSSELLO, ARMANDO, ORLANDINI, ELISABETTA, Armando Rossello   +5 more
core   +1 more source

Exploring Tafamidis Effects Through PBPK–QSP Modelling

Pharmaceutics
Background/Objectives: Tafamidis, a transthyretin kinetic stabilizer, increases circulating transthyretin levels in treated patients. While this effect is well documented, its underlying mechanism remains incompletely understood.
Seweryn Ulaszek, Bartek Lisowski, Barbara Wiśniowska, Sebastian Polak   +3 more
doaj   +1 more source

Investigating transthyretin variants H88R and I107V in amyloid priming: From destabilization to complete dissociation

The FEBS Journal, Volume 293, Issue 15, Page 4680-4703, August 2026.
Investigated mutations in transthyretin (TTR) disrupt the F87‐centered hydrophobic core that stabilizes its tetrameric structure. The mild I107V mutation weakens inter‐chain packing, while H88R fully abolishes tetramer formation, yielding a monomeric, aggregation‐prone form. Structural, biophysical, and computational analyses reveal that both mutations
István L. Bódy, Zsolt Fazekas, Nóra Wágner, Anna J. Kiss‐Szemán, Veronika Harmat, Zoltán Pozsonyi, Dóra K. Menyhárd, András Perczel   +7 more
wiley   +1 more source

Tafamidis for the Treatment of Hereditary Transthyretin Amyloid Cardiomyopathy: A Case Report

, 2017
Tafamidis meglumine is a novel medicine that has been shown to slow the progression of peripheral neurological impairment in patients with hereditary transthyretin amyloidosis (ATTR).
Takayuki Inomata, Yuichirou Iida, Toyoji Kaida, Shunsuke Ishii, Junya Ako, Eiji Kitamura, Emi Maekawa, Toshimi Koitabashi, Yuki Ikeda, Takashi Naruke, Teppei Fujita, Yoshiki Sekijima, Takeru Nabeta   +12 more
core   +1 more source

Long-term tafamidis efficacy in patients with transthyretin amyloid cardiomyopathy by baseline left ventricular ejection fraction [PDF]

Publisher Copyright: © 2024 The Author(s). European Journal of Heart Failure published by John Wiley & Sons Ltd on behalf of European Society of Cardiology.Aims: Patients with transthyretin amyloid cardiomyopathy (ATTR-CM) present with diverse left ...
Damy, Thibaud, Drachman, Brian, Wang, Ronnie, Angeli, Franca S., Hanna, Mazen, Garcia-Pavia, Pablo   +5 more
core   +1 more source

Tafamidis Reduces Death and Hospitalization for Acute Heart Failure in Octogenarian Patients With Transthyretin Cardiac Amyloidosis: A Propensity Score–Weighted Cohort Study

Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease
Background Transthyretin cardiac amyloidosis results from the deposition of misfolded amyloid fibrils in cardiac tissue, leading to progressive heart failure.
Louise Zmuda, Amira Zaroui, Mounira Kharoubi, Emmanuelle Boutin, Frédéric Roca, Silvia Oghina, Emmanuel Teiger, Marie Laurent, Florence Canoui‐Poitrine, Thibaud Damy, Amaury Broussier   +10 more
doaj   +1 more source

Real-World Utilization Patterns, Safety, and Efficacy of Tafamidis in Patients With Hereditary Transthyretin Amyloidosis in Japan

Current Therapeutic Research
: Purpose: Patients with hereditary transthyretin (ATTRv) amyloidosis experience progressive degeneration of the somatic and peripheral nervous system that can impair ambulation, autonomy, and quality of life (QOL).
Hiroaki Konishi, PharmD, Hajime Abe, MD, PhD, Noriko Matsumoto, Yutaka Endo, Yoshiki Sekijima, MD, PhD, Mitsuharu Ueda, MD, PhD, Yukio Ando, MD, PhD   +6 more
doaj   +1 more source