Results 81 to 90 of about 6,309 (194)

Safety and Efficacy of Tafamidis in Chinese Patients with Transthyretin Amyloid Cardiomyopathy

Cardiology and Therapy
Introduction Tafamidis is approved in many countries for the treatment of patients with transthyretin amyloid cardiomyopathy (ATTR-CM). Approval is largely based on findings from an international phase 3 trial.
Zhuang Tian, Daoquan Peng, Wei Ma, Jiangtao Yan, Jian’an Wang, Yida Tang, Wei Jin, Ying Liu, Caiping Jia, Yingxu Gao, Yankun Gong, Xiaohong Sun, Naihan Chen, Shuiqing Zhu, Shuyang Zhang   +14 more
doaj   +1 more source

Evaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells

, 2017
Familial amyloid polyneuropathy (FAP) is caused by mutations of the transthyretin (TTR) gene, predominantly expressed in the liver. Two compounds that knockdown TTR, comprising a small interfering RNA (siRNA; ALN-TTR-02) and an antisense oligonucleotide (
Ackermann, E.J. (Elisabeth), Avsar, Y. (Yesim), Chandhok, G. (Gursimran), Fleischhauer, L. (Lutz), Gollob, J. (Jared), Guo, S. (Shuling), Guttmann, S. (Sarah), Monia, B.P. (Brett), Niemietz, C. (Christoph), Sauer, V.D. (Vanessa), Schmidt, H. (Hartmut), Stella, J. (Jaqueline), Zibert, A. (Andree)   +12 more
core   +1 more source

A Sustainable Visible Light‐Mediated Synthesis of Benzoxazole 2‐Carboxylates/Carboxamides

Chemistry – A European Journal, Volume 31, Issue 68, December 4, 2025.
An efficient photochemical protocol for the synthesis of 2‐ester or 2‐amide benzoxazoles was developed, utilizing 1,8‐dihydroxyanthraquinone and CuI as the photocatalytic system under LED 427 nm irradiation. Furthermore, 2‐ester/amide benzoxazoles serve as intermediates for the synthesis of potentially valuable compounds.
Anna‐Dimitra D. Gerogiannopoulou, Olga G. Mountanea, Maria A. Theodoropoulou, Christoforos G. Kokotos, George Kokotos   +4 more
wiley   +1 more source

Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years

Neurology and Therapy, 2020
Introduction The effects of tafamidis on mortality in Val30Met and non-Val30Met patients with transthyretin amyloidosis with polyneuropathy (ATTR-PN) were evaluated.
Giampaolo Merlini, Teresa Coelho, Márcia Waddington Cruz, Huihua Li, Michelle Stewart, Ben Ebede   +5 more
doaj   +1 more source

Molecular dynamics simulation study of AG10 and tafamidis binding to the Val122Ile transthyretin variant

Biochemistry and Biophysics Reports, 2020
Molecular dynamics (MD) simulations were used to investigate the binding of four ligands to the Val122Ile mutant of the protein transthyretin. Dissociation, misfolding, and subsequent aggregation of mutated transthyretin proteins are associated with the ...
Kevin F. Morris, Riley M. Geoghegan, Emily E. Palmer, Matthew George, Jr., Yayin Fang   +4 more
doaj   +1 more source

Pre-symptomatic testing for neurodegenerative disorders: Middle- to long-term psychopathological impact: Pruebas pre-sintomáticas de enfermedades neurodegenerativas: el impacto psicopatológico a largo plazo [PDF]

, 2017
http://sherpa.ac.uk/romeo/issn/0214-9915/Background. Over the past 20 years, studies have revealed that the communication of a pre-symptomatic test (PST) result for autosomal dominant late-onset diseases, such as Huntington's disease (HD), doesn’t cause ...
Dinis, Maria Alzira Pimenta, Leite, Ângela, Lêdo, Susana, Sequeiros, Jorge, Souto, Teresa   +4 more
core   +1 more source

Evolving Real‐World Data and Evidence Use for New Drugs and Regenerative Medical Products Approvals in Japan—An Analysis of the 6‐Year Trend

Clinical Pharmacology &Therapeutics, Volume 118, Issue 6, Page 1405-1421, December 2025.
Several regulatory initiatives have been made to clarify the acceptability and requirements of real‐world data and real‐world evidence (RWD/E) for the benefit/risk assessment of new medical products in Japan. The objectives of this review were to characterize the use of RWD/E in regulatory applications of new medical products and to describe the ...
Suguru Okami, Kako Shimotsumagari, Yasunari Sadatsuki   +2 more
wiley   +1 more source

A new staging system for hereditary transthyretin amyloidosis in the era of specific amyloidosis therapies

ESC Heart Failure, Volume 12, Issue 6, Page 4125-4139, December 2025.
The aim of our study was to develop a more accurate staging system for hereditary ATTR patients currently receiving specific treatment. A staging system was created using as criteria: GLS ≤ −11%, NT‐proBNP ≤ 2,000 ng/L, and eGFR ≤ 65 mL/min. We managed to accurately stratify patients into three disease stages, significantly different in terms of ...
Gabriela Neculae, Amira Zaroui, Robert Adam, Mounira Kharoubi, Benoit Funalot, Daniel Coriu, Ruxandra Jurcut, Thibaud Damy   +7 more
wiley   +1 more source

Tafamidis therapy in transthyretin amyloid cardiomyopathy: a narrative review from clinical trials and real-world evidence

The Egyptian Heart Journal
Background Amyloidosis is a heterogeneous group of disorders caused by the extracellular deposition of insoluble misfolded proteins, leading to end-organ damage.
Ikponmwosa Jude Ogieuhi, Oshomoh Mark-Anthony Ugiomoh, Kudzaishe Muzofa, Kristen Callender, Johnson David Ayodeji, Nnokam Prayer Nnekachi, Barkavi Thiyagarajan, Emmanuel Obokhai Uduigwome, Abhay Kapoor, Moses Chukwuebuka Odoeke, Reem Gamaleldin Hassan Mohamed, Courage Idahor   +11 more
doaj   +1 more source

Incidence and Predictors of Right Ventricular Reverse Remodeling in Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis

Biomedicines
Background/Objectives: In patients with transthyretin amyloid cardiomyopathy (ATTR-CM), the effect of tafamidis on right ventricular (RV) dysfunction has been poorly investigated.
Nicoleta Nita, Dominik Felbel, Michael Paukovitsch, Felix von Sanden, Elene Walter, Rima Melnic, Wolfgang Rottbauer, Dominik Buckert, Johannes Mörike   +8 more
doaj   +1 more source