Results 291 to 300 of about 61,872 (307)

RNA-targeting and gene editing therapies for transthyretin amyloidosis

Nature Reviews Cardiology, 2022
Alberto Aimo, Vincenzo Castiglione, Maria Franzini   +2 more
exaly   +2 more sources

Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease

Nature Reviews Neurology, 2019
David Adams, Haruki Koike
exaly   +2 more sources

Prevalence and clinical outcomes of transthyretin amyloidosis: a systematic review and meta‐analysis

European Journal of Heart Failure, 2022
Systematic evidence on the prevalence and clinical outcome of transthyretin amyloidosis (ATTR) is missing. We explored: (i) the prevalence of cardiac amyloidosis in various patient subgroups, (ii) survival estimates for ATTR subtypes, and (iii) the ...
A. Antonopoulos, I. Panagiotopoulos, A. Kouroutzoglou, G. Koutsis, P. Toskas, G. Lazaros, K. Toutouzas, D. Tousoulis, K. Tsioufis, C. Vlachopoulos   +9 more
semanticscholar   +1 more source

Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy.

New England Journal of Medicine
BACKGROUND Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, fatal disease. Vutrisiran, a subcutaneously administered RNA interference therapeutic agent, inhibits the production of hepatic transthyretin.
M. Fontana, J. Berk, J. Gillmore, Ronald M. Witteles, M. Grogan, B. Drachman, Thibaud Damy, Pablo Garcia-Pavia, Jorg Taubel, Scott D. Solomon, Farooq H. Sheikh, Nobuhiro Tahara, J. González-Costello, Kenichi Tsujita, C. Morbach, Zoltán Pozsonyi, M. C. Petrie, D. Delgado, P. van der Meer, Andrew Jabbour, A. Bondue, Darae Kim, Olga Azevedo, Steen Hvitfeldt Poulsen, Ali Yilmaz, Ewa A. Jankowska, Vincent Algalarrondo, Andrew Slugg, Pushkal P. Garg, Katherine L. Boyle, E. Yureneva, Nancy Silliman, Lilli Yang, Jihong Chen, S. Eraly, J. Vest, M. Maurer   +36 more
semanticscholar   +1 more source

Cardiac Amyloidosis Due to Transthyretin Protein: A Review.

Journal of the American Medical Association (JAMA)
Importance Systemic amyloidosis from transthyretin (ATTR) protein is the most common type of amyloidosis that causes cardiomyopathy. Observations Transthyretin (TTR) protein transports thyroxine (thyroid hormone) and retinol (vitamin A) and is ...
F. Ruberg, Mathew S. Maurer
semanticscholar   +1 more source

Vutrisiran: A Review in Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis

Drugs, 2023
Tina Nie, Young-A. Heo, M. Shirley
semanticscholar   +1 more source

SGLT2 Inhibitor Therapy in Patients With Transthyretin Amyloid Cardiomyopathy.

Journal of the American College of Cardiology
A. Porcari, F. Cappelli, C. Nitsche, D. Tomasoni, G. Sinigiani, S. Longhi, Luca Bordignon, Ahmad Masri, Matteo Serenelli, M. Urey, B. Musumeci, A. Cipriani, Marco Canepa, R. Badr-Eslam, C. Kronberger, Cristina Chimenti, M. Zampieri, Valentina Allegro, Y. Razvi, R. Patel, A. Ioannou, M. Rauf, A. Petrie, C. Whelan, M. Emdin, M. Metra, Marco Merlo, G. Sinagra, P. Hawkins, S. Solomon, J. Gillmore, M. Fontana   +31 more
semanticscholar   +1 more source

Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

Lancet Neurology, The, 2021
Jonas Wixner, Arnt V Kristen, John L Berk   +2 more
exaly  

Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial

Amyloid: the International Journal of Experimental and Clinical Investigation: the Official Journal of the International Society of Amyloidosis, 2023
David Adams, Mark Taylor, Violaine Planté-Bordeneuve   +2 more
exaly  

Transthyretin Amyloid Cardiomyopathy

Journal of the American College of Cardiology, 2019
Frederick L Ruberg, Martha Grogan, Jeffery W Kelly   +2 more
exaly