Results 31 to 40 of about 231,717 (193)

Long-Term Survival With Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy [PDF]

, 2021
BACKGROUND: Tafamidis is approved in many countries for the treatment of transthyretin amyloid cardiomyopathy. This study reports data on the long-term efficacy of tafamidis from an ongoing long-term extension (LTE) to the pivotal ATTR-ACT (Tafamidis in ...
Sultan, MB, Hoffman, JE, Hummel, SL, Gundapaneni, B, Elliott, P, Lenihan, DJ, Ebede, B, Shah, SJ, Drachman, BM, Gottlieb, SS, Li, B   +10 more
core   +2 more sources

Technical Feasibility of Implementing and Commercializing a Machine Learning Model for Rare Disease Prediction

IEEE Access, 2023
Wild-type transthyretin amyloid cardiomyopathy is an under-recognized cause of heart failure. Pfizer previously developed a machine learning model that performed well in identifying wild-type transthyretin amyloid cardiomyopathy vs.
George Koutitas, Kimberly Nolen, Sepideh Attal, Anastasios Ventouris, Yinnon Dolev, Hans Thijs Van Den Broek   +5 more
doaj   +1 more source

Temporal Trends of Wild-Type Transthyretin Amyloid Cardiomyopathy in the Transthyretin Amyloidosis Outcomes Survey [PDF]

, 2021
Background: Transthyretin amyloid cardiomyopathy results from the accumulation of wild-type (ATTRwt) or variant (ATTRv) transthyretin amyloid fibrils in the myocardium.
Steidley D., Plante-Bordeneuve V., Costello J. G., Slosky D., Mueller C., Sarswat N., Garcia-Pavia P., Murali S., Wixner J., Polydefkis M., Siu A., Sekijima Y., Witteles R., Gess B., Marburger T., Cirami C. L., Hummel S., Emdin M., Lenihan D., Jacoby D., Kristen A. V., LoRusso S., Nativi-Nicolau J., Lairez O., Luigetti M., Adams D., Maurer M. S., Gonzalez Duarte Briseno M. A., Tauras J., Waddington-Cruz M., Moreno J. G., Azevedo O., Hoffman J., Rapezzi C., Schmidt H., Luo J., Drachman B., Nienhuis H., Moelgaard H., Dispenzieri A., Shah S., Cotts W., Gottlieb S., Barroso F. A., Van Cleemput J., Zivkovic S., Jeon E. -S., Ralph J., Amass L., Ventura H., Grogan M., Quan D., Vita G., Tallaj J., Parman Y., Inamo J., Fine N., Campistol Plana J. M., Yamashita T., Chapman D.   +59 more
core   +3 more sources

Tafamidis in Transthyretin Amyloid Cardiomyopathy [PDF]

Circulation: Heart Failure, 2015
Background— Transthyretin (TTR) amyloidosis is a progressive systemic disorder caused by misfolded TTR monomers that cumulatively deposit in the heart and systemically as amyloid. Methods and Results— This phase 2 open-label trial evaluated the stabilization of TTR tetramers using ...
Mathew S, Maurer, Donna R, Grogan, Daniel P, Judge, Rajiv, Mundayat, Jeff, Packman, Ilise, Lombardo, Arshed A, Quyyumi, Janske, Aarts, Rodney H, Falk   +8 more
openaire   +2 more sources

Progress in New Therapies Targeting the Pathogenesis of Cardiomyopathywith Hypertrophic Phenotype

罕见病研究, 2023
Hypertrophic cardiomyopathy (HCM) is cardiomyopathy with a clinical phenotype of cardiac hypertrophy. The etiology includes genetically defective encoding sarcomeres, congenital metabolic diseases such as lysosomal storage diseases, systemic amyloidosis ...
LIU Yanbo, TIAN Zhuang, ZHANG Shuyang
doaj   +1 more source

Transthyretin Stabilizers and Seeding Inhibitors as Therapies for Amyloid Transthyretin Cardiomyopathy

Pharmaceutics, 2023
Transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM) is a progressive and increasingly recognized cause of heart failure which is associated with high mortality and morbidity. ATTR-CM is characterized by the misfolding of TTR monomers and their deposition within the myocardium as amyloid fibrils.
Paolo Morfino, Alberto Aimo, Giuseppe Vergaro, Chiara Sanguinetti, Vincenzo Castiglione, Maria Franzini, Marco Alfonso Perrone, Michele Emdin   +7 more
openaire   +7 more sources

Natriuretic Peptide and Transthyretin and Their Relationship With Clinical Outcomes: Results From ATTRIBUTE‐CM, a Randomized Controlled Trial

Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease
Background Transthyretin amyloid cardiomyopathy (ATTR‐CM) carries high risk for all‐cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular hospitalization (CVH). NT‐proBNP (N‐terminal pro‐B‐type natriuretic peptide) and serum TTR (sTTR)
Onyedika John Ilonze, Daniel Philip Judge, Ahmad Masri, Prem Soman, Francesco Cappelli, Michel Georges Khouri, Nitasha Sarswat, Mazen Hanna, Yuxi Liu, Gillian Murtagh, Kuangnan Xiong, Adam Castaño, Jean‐François Tamby, Jonathan C. Fox, Uma Sinha, Jan Marie Griffin, Julian D. Gillmore, James Louis Januzzi   +17 more
doaj   +1 more source

Transthyretin amyloid cardiomyopathy

, 2020
Transthyretin amyloid cardiomyopathy (ATTR-CM) resulting from deposition of transthyretin amyloid fibrils in the heart is an underrecognised cause of heart failure in the elderly and is associated with a poor life expectancy.
Hansen, Peter Riis, Krakauer, Martin
core   +1 more source

Transthyretin amyloid cardiomyopathy: Literature review and red‐flag symptom clusters for each cardiology specialty

ESC Heart Failure, Volume 12, Issue 2, Page 955-967, April 2025.
Abstract Wild‐type transthyretin amyloid cardiomyopathy (ATTRwt‐CM) is a progressive and infiltrative cardiac disorder that may cause fatal consequences if left untreated. The estimated survival time from diagnosis is approximately 3–6 years. Because of the non‐specificity of initial symptom manifestation and insufficient awareness among treating ...
Yasuhiro Izumiya, Toru Kubo, Jin Endo, Seiji Takashio, Masatoshi Minamisawa, Jun Hamada, Tomonori Ishii, Hajime Abe, Hiroaki Konishi, Kenichi Tsujita   +9 more
wiley   +1 more source

Prevalence of transthyretin cardiac amyloidosis in undifferentiated heart failure with preserved ejection fraction

ESC Heart Failure, Volume 12, Issue 2, Page 1176-1182, April 2025.
Abstract Aims Transthyretin amyloid cardiomyopathy (ATTR‐CM) is an increasinglyrecognized cause of heart failure with preserved ejection fraction (HFpEF), which may be diagnosed non‐invasively using 99mTc 3,3‐diphosphono‐1,2‐propanodicarboxylic acid (DPD) scintigraphy‐based diagnostic criteria.
L. Healy, G. Giblin, A. Gray, N. Starr, L. Murphy, D. O'Sullivan, E. Kavanagh, C. Howley, C. Tracey, E. Morrin, A. McDaid, A. Clarke, J.O. O'Neill, E. Joyce, M. O'Connell, N. G. Mahon   +15 more
wiley   +1 more source