Results 61 to 70 of about 6,309 (194)

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

Risk perception in subjects at-risk for Familial Amyloidotic Polyneuropathy: Percepción del riesgo en sujetos con riesgo de Polineuropatía Amiloide Familiar [PDF]

, 2017
http://sherpa.ac.uk/romeo/issn/2011-2777/The aims of this study are to know if subjects at-risk were aware of their 50% risk for Familial Amyloidotic Polyneuropathy (FAP); to know the value of the subjective risk; to understand the association between ...
Dinis, Maria Alzira Pimenta, Leite, Ângela, Paúl, Constança, Sequeiros, Jorge   +3 more
core   +2 more sources

Serum Neurofilament Light Chain and Glial Fibrillary Acidic Protein as Biomarkers in Hereditary Transthyretin Amyloidosis Polyneuropathy

Journal of the Peripheral Nervous System, Volume 31, Issue 1, March 2026.
ABSTRACT Background and Aims In individuals with hereditary transthyretin amyloidosis (ATTRv) polyneuropathy, monitoring of disease progression and treatment response is crucial. The objective is to determine if serum neurofilament light chain (sNfL) and serum glial fibrillary acidic protein (sGFAP) are reliable biomarkers of ATTRv polyneuropathy ...
Valentin Loser, Pascal Benkert, Alex Vicino, Nicolas Ghika, Pansy Lim Dubois Ferrière, Chantal Daigneault, Thierry Kuntzer, Aleksandra Maleska Maceski, Jens Kuhle, Marie Théaudin   +9 more
wiley   +1 more source

Impact of tafamidis on myocardial function and CMR tissue characteristics in transthyretin amyloid cardiomyopathy

ESC Heart Failure
Aims Tafamidis improves clinical outcomes in transthyretin amyloid cardiomyopathy (ATTR‐CM), yet how tafamidis affects cardiac structure and function remains poorly described.
Stephan Dobner, Benedikt Bernhard, Lorenz Ninck, Monika Wieser, Adam Bakula, Andreas Wahl, Valentin Köchli, Giancarlo Spano, Martina Boscolo Berto, Elena Elchinova, Yasaman Safarkhanlo, Stefan Stortecky, Jonathan Schütze, Isaac Shiri, Lukas Hunziker, Christoph Gräni   +15 more
doaj   +1 more source

An acridine derivative, [4,5-bis{(N-carboxy methyl imidazolium)methyl}acridine] dibromide, shows anti-TDP-43 aggregation effect in ALS disease models [PDF]

, 2016
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease associated with aggregation of TAR DNA-binding protein-43 (TDP-43) in neuronal cells and manifests as motor neuron dysfunction & muscle atrophy.
G, Prabusankar, Girdhar, A, Patel, Basant Kumar, Prasad, A, Raju, G, Sivalingam, V, Taneja, V, Vats, A, Verma, M   +8 more
core   +1 more source

Baseline Characteristics and Secondary Medication Adherence Patterns Among Patients Receiving Tafamidis Prescriptions: A Retrospective Analysis Using a National Specialty Pharmacy Dispensing Database

Patient Preference and Adherence, 2022
Anuja Roy,1 Andrew Peterson,2 Nick Marchant,1 Jose Alvir,3 Rahul Bhambri,4 Jason Lynn,4 Darrin Benjumea,5 Sapna Prasad,6 Alex O’Brien,6 Yong Chen,7 Jason Kemner,8 Bhash Parasuraman8 1Global HEOR, Patient & Health Impact, Rare Diseases BU, Pfizer Inc, New
Roy A, Peterson A, Marchant N, Alvir J, Bhambri R, Lynn J, Benjumea D, Prasad S, O'Brien A, Chen Y, Kemner J, Parasuraman B   +11 more
doaj  

Comparative Outcomes of a Transthyretin Amyloid Cardiomyopathy Cohort Versus Patients With Heart Failure With Preserved Ejection Fraction Enrolled in the TOPCAT Trial

Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease, 2023
Background Transthyretin cardiac amyloidosis (ATTR‐CM), found in 6% to 15% of cohorts with heart failure with preserved ejection fraction, has long been considered a rare disease with poor prognosis.
Morris M. Kim, Mark Prasad, Yunwoo Burton, Clinton M. Kolseth, Yuanzi Zhao, Pranav Chandrashekar, Babak Nazer, Ahmad Masri   +7 more
doaj   +1 more source

Efficacy and safety of tafamidis doses in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and long-term extension study. [PDF]

, 2020
Aims Tafamidis is an effective treatment for transthyretin amyloid cardiomyopathy (ATTR-CM) in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT).
Damy, Thibaud, García Pavía, Pablo, Gundapaneni, Balarama, Hanna, Mazen, Judge, Daniel P., Merlini, Giampaolo, Patterson, Terrell A., Riley, Steven, Schwartz, Jeffrey H., Sultan, Marla B., Witteles, Ronald   +10 more
core   +1 more source

Tafamidis 61 mg Patient Characteristics and Persistency? A Retrospective Analysis of German Statutory Health Insurance Data (IQVIA™ LRx)

Cardiology and Therapy
Introduction Tafamidis is the first drug approved by the European Commission for the treatment of wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related ...
Sepideh Attal, Jason Kemner, Jose Alvir, Sebastian Barth, Sofia Schuessler   +4 more
doaj   +1 more source

Amyloid seeding of transthyretin by ex vivo cardiac fibrils and its inhibition [PDF]

, 2018
Each of the 30 human amyloid diseases is associated with the aggregation of a particular precursor protein into amyloid fibrils. In transthyretin amyloidosis (ATTR), mutant or wild-type forms of the serum carrier protein transthyretin (TTR), synthesized ...
Benson, Merrill D., Chung, Kevin, Cohn, Whitaker, Eisenberg, David S., Lee, Ji H., Saelices, Lorena, Whitelegge, Julian P.   +6 more
core   +1 more source