Results 21 to 30 of about 5,684 (155)

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).
Hanna, M., Schwartz, J. H., Garcia-Pavia, P., Sultan, Marla B., Mazen Hanna, Damy, Thibaud, Daniel P. Judge, Merlini, G., Patterson, T. A., García Pavía, Pablo, Gundapaneni, Balarama, Damy, T., Ronald Witteles, Balarama Gundapaneni, Judge, D. P., Riley, S., Witteles, R., Gundapaneni, B., Riley, Steven, Giampaolo Merlini, Hanna, Mazen, Judge, Daniel P., Marla B. Sultan, Terrell A. Patterson, Patterson, Terrell A., Thibaud Damy, Pablo Garcia‐Pavia, Witteles, Ronald, Steven Riley, Schwartz, Jeffrey H., Jeffrey H. Schwartz, Merlini, Giampaolo, Sultan, M. B., Judge, Daniel P, Patterson, Terrell A, Garcia-Pavia, Pablo, Schwartz, Jeffrey H, Sultan, Marla B   +37 more
core   +1 more source

Five-year Survival With Tafamidis In Patients With Transthyretin Amyloid Cardiomyopathy

, 2022
Tafamidis was shown to significantly reduce mortality in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and is approved for the treatment of patients with transthyretin amyloid cardiomyopathy (ATTR-CM).
Sultan, Marla B, Elliott, Perry, Gundapaneni, Balarama, Hoffman, James E, Perry Elliott, Balarama Gundapaneni, James E. Hoffman, Marla B. Sultan   +7 more
core   +1 more source

Tafamidis is entering the clinical arena for the treatment of transthyretin‐related cardiomyopathy: certainties and unmet needs

, 2021
This article refers to ‘Efficacy and safety of tafamidis doses in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and long-term extension study’ by T.
Rapezzi C., Aimo A., Emdin M., Alberto Aimo, Michele Emdin, Claudio Rapezzi   +5 more
core   +1 more source

Contemporary real-world treatment of cardiac transthyretin amyloidosis with tafamidis: a long-term multicentre study. [PDF]

ESC Heart Fail
Aims Tafamidis was the first drug approved for transthyretin amyloid cardiomyopathy (ATTR-CM), but contemporary real-world evidence is lacking. Methods and results
Nies RJ, Ney S, Kindermann I, Bewarder Y, Zimmer A, Knebel F, Hahn K, Spethmann S, Luedike P, Michel L, Rassaf T, Papathanasiou M, Störk S, Cejka V, Polzin A, Voß F, Unsöld B, Meindl C, Paulus M, Yilmaz A, Chamling B, Morbach C, Pfister R.   +22 more
europepmc   +2 more sources

Tafamidis decreased cardiac amyloidosis deposition in patients with Ala97Ser hereditary transthyretin cardiomyopathy: a 12-month follow-up cohort study

Orphanet Journal of Rare Diseases, 2023
Background Transthyretin cardiac cardiomyopathy (ATTR-CM) is a rare but life-threatening disease. Tafamidis is an effective treatment for patients with ATTR-CM, however its long-term effects on cardiac remodeling and cardiac amyloid deposition are ...
Cheng-Hsuan Tsai, Chi-Chao Chao, Sung-Tsang Hsieh, An-Li Yu, Yuan-Kun (Aden) Wu, Mei-Fang Cheng, Ming-Jen Lee, Chia-Hung Chou, Chia-Tung Shun, Hsueh-Wen Hsueh, Jimmy Jyh-Ming Juang, Ping-Huei Tseng, Mao-Yuan Su, Yen-Hung Lin   +13 more
doaj   +1 more source

Thermal and Structural Characterization of Two Crystalline Polymorphs of Tafamidis Free Acid

Molecules, 2022
Tafamidis, chemical formula C14H7Cl2NO3, is a drug used to delay disease progression in adults suffering from transthyretin amyloidosis, and is marketed worldwide under different tradenames as a free acid or in the form of its meglumine salt.
Norberto Masciocchi, Vincenzo Mirco Abbinante, Marco Zambra, Giuseppe Barreca, Massimo Zampieri   +4 more
doaj   +1 more source

Evaluation of the Current ATTR-CM Treatment Landscape via a Mathematical Model of TTR Dissociation and Amyloid Formation. [PDF]

CPT Pharmacometrics Syst Pharmacol
ABSTRACT Transthyretin amyloid cardiomyopathy (ATTR‐CM) is a progressive, often fatal disease arising from the dissociation of circulating transthyretin (TTR) tetramers into monomers that misfold and form amyloid fibrils that deposit in the myocardium and other tissues. Approved treatment paradigms involve tetramer stabilization with small molecules or
Methven Jeknić S, Ahn-Horst TA, Ji AX, Ju JH, Paull M, Ji SG, Sinha U.   +6 more
europepmc   +2 more sources

Brain Permeable Tafamidis Amide Analogs for Stabilizing TTR and Reducing APP Cleavage

, 2020
Tafamidis, 1, a potent transthyretin kinetic stabilizer, weakly inhibits the γ-secretase enzyme in vitro. We have synthesized four amide derivatives of 1.
Anjana Sinha (2465398), Younhee Cho, Subhash C. Sinha, Jerry C. Chang (2148970), Katherina Gindinova (6467117), Peng Xu (17962), Jeffery W. Kelly (185563), Peng Xu, Yue Ming Li (8565660), Paul Greengard, Jerry C. Chang, Katherina Gindinova, Xianzhong Wu (298055), Yue Ming Li, Jeffery W. Kelly, Paul Greengard (199705), Subhash C. Sinha (1574209), Weilin Sun (214818), Anjana Sinha, Xianzhong Wu, Younhee Cho (806021), Weilin Sun   +21 more
core   +2 more sources

Biophysical characterization and modulation of Transthyretin Ala97Ser

Annals of Clinical and Translational Neurology, 2019
Objective Ala97Ser (A97S) is the major transthyretin (TTR) mutation in Taiwanese patients of familial amyloid polyneuropathy (FAP), characterized by a late‐onset but rapidly deteriorated neuropathy.
Yo‐Tsen Liu, Yueh‐Jung Yen, Frans Ricardo, Yu Chang, Pei‐Hao Wu, Shing‐Jong Huang, Kon‐Ping Lin, Tsyr‐Yan Yu   +7 more
doaj   +1 more source

Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy [PDF]

, 2018
BACKGROUND: Transthyretin amyloid cardiomyopathy is caused by the deposition of transthyretin amyloid fibrils in the myocardium. The deposition occurs when wild-type or variant transthyretin becomes unstable and misfolds. Tafamidis binds to transthyretin,
Gundapaneni, B, Merlini, G, Grogan, M, Kristen, AV, Elliott, PM, Schwartz, JH, Rapezzi, C, Witteles, R, Hanna, M, Judge, DP, Sultan, MB, Maurer, MS, Barsdorf, AI, Damy, T, Schumacher, J, Waddington-Cruz, M, Huber, P, Riley, S, Stewart, M, Shah, SJ, Drachman, BM, Patterson, TA   +21 more
core   +2 more sources