Results 71 to 80 of about 5,684 (155)

Influence of baseline neurologic severity on disease progression and the associated disease-modifying effects of tafamidis in patients with transthyretin amyloid polyneuropathy

Orphanet Journal of Rare Diseases, 2018
Background Emerging evidence suggests that several factors can impact disease progression in transthyretin amyloid polyneuropathy (ATTR-PN). The present analysis used longitudinal data from Val30Met patients participating in the tafamidis (selective TTR ...
Leslie Amass, Huihua Li, Balarama K. Gundapaneni, Jeffrey H. Schwartz, Denis J. Keohane   +4 more
doaj   +1 more source

Predictive model of response to tafamidis in hereditary ATTR polyneuropathy [PDF]

, 2019
BACKGROUNDThe hereditary transthyretin (TTR) amyloidoses are a group of diseases for which several disease-modifying treatments are now available. Long-term effectiveness of these therapies is not yet fully known.
Anselmo, J., Novais, M., Fernandes, J., Rodrigues, C., Brighty, G., Powers, E., Kelly, J., Coelho, T., Mesgazardeh, J., Powers, D., Monteiro, C.   +10 more
core   +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

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

Determining the selectivity of TBBPA and tafamidis for TTR in human plasma.

, 2016
TBBPA and tafamidis were titrated at different concentrations in human plasma from a single healthy donor and incubated for 2 h. The dissociation of plasma TTR was initiated by the addition of urea at a final concentration of 4.0 M.
Afshan Begum (746273), Patrik L. Andersson (618256), Kristoffer Brännström (343000), Lina Nilsson (1541728), Jin Zhang (53297), Irina Iakovleva (533699), Alexandra Wijsekera (2616667), Anders Olofsson (343002), A. Elisabeth Sauer-Eriksson (338132)   +8 more
core   +1 more source

Clinical phenotype and prognosis of real-world patients with wild-type transthyretin amyloid cardiomyopathy treated with tafamidis [PDF]

Aims: Tafamidis reshaped the treatment paradigm in transthyretin amyloid cardiomyopathy (ATTR-CM) based on a phase-3 randomized controlled trial, but real-world data on its use remain limited.
Porcari, Aldostefano, Emdin, Michele, Foti, Grazia, Sanna, Giuseppe Damiano, Sena, Giuseppe, Gardini, Elisa, Colio, Federica, D'Elia, Emilia, Pozzan, Marco, Marini, Marco, De Michieli, Laura, Tini, Giacomo, Di Bella, Gianluca, Vagnarelli, Fabio, Canepa, Marco, Monda, Emanuele, Perfetto, Federico, Biagini, Elena, Merlo, Marco, Bonacchi, Giacomo, Driussi, Mauro, Serenelli, Matteo, Zanoletti, Margherita, Palladini, Giovanni, Cantone, Anna, Sturdà, Francesca, Limongelli, Giuseppe, Sinigiani, Giulio, Merlini, Giampaolo, Cappelli, Francesco, Vergaro, Giuseppe, Metra, Marco, Carigi, Samuela, Ruotolo, Irene, Lofiego, Carla, Sinagra, Gianfranco, Tomasoni, Daniela, Perlini, Stefano, Cipriani, Alberto, Gentile, Piero, Aimo, Alberto, Marazia, Stefania, Longhi, Simone, Bartolotti, Michela, Musumeci, Beatrice, Imazio, Massimo, Di Marco, Massimo, Musca, Francesco, Milani, Paolo   +48 more
core   +1 more source

Role of ^99mTc-pyrophosphate myocardial scintigraphy in assessing the efficiency of tafamidis therapy: a case series

Российский кардиологический журнал
Introduction. In recent years, the detection rate of transthyretin amyloid cardiomyopathy has been rapidly increasing. The only drug registered as a pathogenetic therapy in Russia is tafamidis.
A. A. Shoshina, A. A. Ansheles, S. N. Nasonova, I. V. Zhirov, V. B. Sergienko, S. N. Tereshchenko   +5 more
doaj   +1 more source

Efficacy of Tafamidis in Patients With Hereditary and Wild-Type Transthyretin Amyloid Cardiomyopathy: Further Analyses From ATTR-ACT [PDF]

, 2020
BACKGROUND: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, fatal disorder resulting from mutations in the transthyretin gene (ATTRv) or the deposition of denatured wild-type transthyretin (ATTRwt).
Sultan, MB, Berk, JL, Velazquez, EJ, Boman, K, Maurer, MS, Damy, T, Gundapaneni, B, Elliott, P, Patterson, TA, Schwartz, JH, Rapezzi, C, Nativi-Nicolau, J   +11 more
core   +1 more source

Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Variant Transthyretin Amyloid Cardiomyopathy

Cardiology and Therapy
Introduction Transthyretin amyloidosis (ATTR) is a progressive, heterogeneous rare disease manifesting as ATTR polyneuropathy (ATTR-PN), ATTR cardiomyopathy (ATTR-CM), or a mixed phenotype. Tafamidis meglumine (20 mg po qd) is approved in some markets to
Nicholas Streicher, Leslie Amass, Rong Wang, Jennifer M. Stephens, Traci LeMasters, Rutika Raina, Emma Merrill, Farooq H. Sheikh   +7 more
doaj   +1 more source

Early intervention with tafamidis provides long-term (5.5-year) delay of neurologic progression in transthyretin hereditary amyloid polyneuropathy

, 2017
Transthyretin hereditary amyloid polyneuropathy, also traditionally known as transthyretin familial amyloid polyneuropathy (ATTR-FAP), is a rare, relentless, fatal hereditary disorder.
Jeffrey Schwartz (42024), Balarama Gundapaneni (2919245), Huihua Li (166588), Leslie Amass (2919239), Márcia Waddington Cruz (2919242), Denis Keohane (2919236)   +5 more
core   +1 more source