Results 331 to 336 of about 200,281 (336)

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

Systemic Amyloidosis Recognition, Prognosis, and Therapy: A Systematic Review.

Journal of the American Medical Association (JAMA), 2020
Importance Many patients with systemic amyloidosis are underdiagnosed. Overall, 25% of patients with immunoglobulin light chain (AL) amyloidosis die within 6 months of diagnosis and 25% of patients with amyloid transthyretin (ATTR) amyloidosis die within
M. Gertz, A. Dispenzieri
semanticscholar   +1 more source

RNA-targeting and gene editing therapies for transthyretin amyloidosis

Nature Reviews Cardiology, 2022
Alberto Aimo, V. Castiglione, C. Rapezzi, M. Franzini, G. Panichella, G. Vergaro, J. Gillmore, M. Fontana, C. Passino, M. Emdin   +9 more
semanticscholar   +1 more source

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

Nature Reviews Neurology, 2019
D. Adams, H. Koike, M. Slama, T. Coelho
semanticscholar   +1 more source

Systemic immunoglobulin light chain amyloidosis

Nature Reviews Disease Primers, 2018
G. Merlini, A. Dispenzieri, V. Sanchorawala, S. Schönland, G. Palladini, P. Hawkins, M. Gertz   +6 more
semanticscholar   +1 more source