Results 11 to 20 of about 200,281 (336)

Marked progress in AL amyloidosis survival: a 40-year longitudinal natural history study

Blood Cancer Journal, 2021
The recent decades have ushered in considerable advancements in the diagnosis and treatment of systemic light chain (AL) amyloidosis. As disease outcomes improve, AL amyloidosis-unrelated factors may impact mortality. In this study, we evaluated survival
Andrew Staron, Luke Zheng, Gheorghe Doros, Lawreen H. Connors, Lisa M. Mendelson, Tracy Joshi, Vaishali Sanchorawala   +6 more
doaj   +2 more sources

Sex-specific effects of microbiome perturbations on cerebral Aβ amyloidosis and microglia phenotypes. [PDF]

Journal of Experimental Medicine, 2019
We demonstrated that an antibiotic cocktail (ABX)-perturbed gut microbiome is associated with reduced amyloid-β (Aβ) plaque pathology and astrogliosis in the male amyloid precursor protein (APP)SWE /presenilin 1 (PS1)ΔE9 transgenic model of Aβ ...
Baufeld, Caroline, Butovsky, Oleg, Dodiya, Hemraj B, Gilbert, Jack A, Gottel, Neil, Kuntz, Thomas, Leibowitz, Jeffrey, Shaik, Shabana M, Sisodia, Sangram S, Zhang, Xiaoqiong, Zhang, Xulun   +10 more
core   +3 more sources

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.

New England Journal of Medicine, 2021
BACKGROUND Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein in tissues, predominantly the nerves and heart.
J. Gillmore, E. Gane, J. Taubel, J. Kao, M. Fontana, M. Maitland, Jessica Seitzer, D. O’Connell, Kathryn R Walsh, Kristy Wood, Jonathan Phillips, Yuanxin Xu, Adam Amaral, A. Boyd, J. Cehelsky, M. McKee, A. Schiermeier, O. Harari, A. Murphy, C. Kyratsous, B. Zambrowicz, Randy Soltys, D. Gutstein, J. Leonard, L. Sepp-Lorenzino, D. Lebwohl   +25 more
semanticscholar   +1 more source

Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis.

New England Journal of Medicine, 2023
BACKGROUND Transthyretin amyloidosis, also called ATTR amyloidosis, is associated with accumulation of ATTR amyloid deposits in the heart and commonly manifests as progressive cardiomyopathy. Patisiran, an RNA interference therapeutic agent, inhibits the
M. Maurer, P. Kale, M. Fontana, J. Berk, M. Grogan, F. Gustafsson, Rebecca R Hung, R. Gottlieb, Thibaud Damy, Alejandra González-Duarte, N. Sarswat, Yoshiki Sekijima, Nobuhiro Tahara, Mark S. Taylor, M. Kubanek, E. Donal, T. Paleček, Kenichi Tsujita, W. Tang, Wen-Chung Yu, L. Obici, M. Simões, Fábio Fernandes, S. Poulsen, Igor Diemberger, F. Perfetto, S. Solomon, Marcelo F Di Carli, Prajakta Badri, M. White, Jihong Chen, E. Yureneva, M. Sweetser, P. Jay, Pushkal P. Garg, J. Vest, J. Gillmore   +36 more
semanticscholar   +1 more source

Diagnosis and treatment of cardiac amyloidosis: a position statement of the ESC Working Group on Myocardial and Pericardial Diseases.

European Heart Journal, 2021
Cardiac amyloidosis is a serious and progressive infiltrative disease that is caused by the deposition of amyloid fibrils at the cardiac level. It can be due to rare genetic variants in the hereditary forms or as a consequence of acquired conditions ...
P. García-Pavía, C. Rapezzi, Y. Adler, M. Arad, C. Basso, A. Brucato, I. Burazor, A. Caforio, T. Damy, U. Eriksson, M. Fontana, J. Gillmore, E. Gonzalez-Lopez, M. Grogan, S. Heymans, M. Imazio, I. Kindermann, A. Kristen, M. Maurer, G. Merlini, A. Pantazis, S. Pankuweit, A. Rigopoulos, A. Linhart   +23 more
semanticscholar   +1 more source

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

Amyloid: Journal of Protein Folding Disorders, 2022
Background The study objective was to assess the effect of vutrisiran, an RNA interference therapeutic that reduces transthyretin (TTR) production, in patients with hereditary transthyretin (ATTRv) amyloidosis with polyneuropathy.
D. Adams, I. Tournev, Mark S. Taylor, T. Coelho, V. Planté-Bordeneuve, J. Berk, A. González-Duarte, J. Gillmore, S. Low, Y. Sekijima, L. Obici, Chongshu Chen, P. Badri, S. Arum, J. Vest, M. Polydefkis   +15 more
semanticscholar   +1 more source

Amyloid nomenclature 2022: update, novel proteins, and recommendations by the International Society of Amyloidosis (ISA) Nomenclature Committee

Amyloid: Journal of Protein Folding Disorders, 2022
The Nomenclature Committee of the International Society of Amyloidosis met at the XVIII International Symposium on Amyloidosis in September and virtually in October 2022 with discussions resulting in this upgraded nomenclature recommendation.
J. Buxbaum, A. Dispenzieri, D. Eisenberg, M. Fändrich, G. Merlini, M. Saraiva, Y. Sekijima, P. Westermark   +7 more
semanticscholar   +1 more source

Daratumumab-Based Treatment for Immunoglobulin Light-Chain Amyloidosis.

New England Journal of Medicine, 2021
BACKGROUND Systemic immunoglobulin light-chain (AL) amyloidosis is characterized by deposition of amyloid fibrils of light chains produced by clonal CD38+ plasma cells. Daratumumab, a human CD38-targeting antibody, may improve outcomes for this disease.
E. Kastritis, G. Palladini, M. Minnema, A. Wechalekar, A. Jaccard, Hans C. Lee, V. Sanchorawala, S. Gibbs, P. Mollee, C. Venner, Jin-Hai Lu, S. Schönland, M. Gatt, Kenshi Suzuki, Kihyun Kim, M. Cibeira, M. Beksaç, E. Libby, J. Valent, V. Hungria, S. Wong, M. Rosenzweig, N. Bumma, A. Huart, M. Dimopoulos, D. Bhutani, A. Waxman, S. Goodman, J. Zonder, Selay Lam, K. Song, T. Hansen, S. Manier, W. Roeloffzen, K. Jamroziak, F. Kwok, C. Shimazaki, Jin-Seok Kim, E. Crusoé, T. Ahmadi, N. Tran, X. Qin, S. Vasey, B. Tromp, J. Schecter, B. Weiss, S. Zhuang, J. Vermeulen, G. Merlini, R. Comenzo   +49 more
semanticscholar   +1 more source

Diagnosis and treatment of cardiac amyloidosis. A position statement of the European Society of Cardiology Working Group on Myocardial and Pericardial Diseases

European Journal of Heart Failure, 2021
Cardiac amyloidosis is a serious and progressive infiltrative disease that is caused by the deposition of amyloid fibrils at the cardiac level. It can be due to rare genetic variants in the hereditary forms or as a consequence of acquired conditions ...
P. García-Pavía, C. Rapezzi, Y. Adler, M. Arad, C. Basso, A. Brucato, I. Burazor, A. Caforio, T. Damy, U. Eriksson, M. Fontana, J. Gillmore, E. Gonzalez-Lopez, M. Grogan, S. Heymans, M. Imazio, I. Kindermann, A. Kristen, M. Maurer, G. Merlini, A. Pantazis, S. Pankuweit, A. Rigopoulos, A. Linhart   +23 more
semanticscholar   +1 more source

Amyloidosis in Alzheimer’s Disease: Pathogeny, Etiology, and Related Therapeutic Directions

Molecules, 2022
The amyloid hypothesis of Alzheimer’s disease has long been the predominant theory, suggesting that Alzheimer’s disease is caused by the accumulation of amyloid beta protein (Aβ) in the brain, leading to neuronal toxicity in the central nervous system ...
Chen Ma, Fenfang Hong, Shulong Yang
semanticscholar   +1 more source