Results 1 to 10 of about 162,291 (263)

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

The Amyloidosis Forum: a public private partnership to advance drug development in AL amyloidosis [PDF]

Orphanet Journal of Rare Diseases, 2020
Background Immunoglobulin light chain (AL) amyloidosis is a rare, multi-systemic disorder characterized by two disease processes: an underlying plasma cell dyscrasia that provides the source of pathologic light chains, and the resulting organ dysfunction
The Inaugural Amyloidosis Forum Panelists, Isabelle Lousada   +1 more
doaj   +3 more sources

The lung in amyloidosis [PDF]

European Respiratory Review, 2017
Amyloidosis is a disorder caused by misfolding of autologous protein and its extracellular deposition as fibrils, resulting in vital organ dysfunction and eventually death. Pulmonary amyloidosis may be localised or part of systemic amyloidosis. Pulmonary
Paolo Milani, Marco Basset, Francesca Russo, Andrea Foli, Giovanni Palladini, Giampaolo Merlini   +5 more
doaj   +3 more sources

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

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

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

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

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

Combined Subcutaneous Fat Aspirate and Skin Tru-Cut Biopsy for Amyloid Screening in Patients with Suspected Systemic Amyloidosis

Molecules, 2021
Screening for systemic amyloidosis is typically carried out with abdominal fat aspirates with varying reported sensitivities. Fat aspirates are preferred for use in primary screening instead of organ biopsies as they are less invasive and thereby ...
Charlotte Toftmann Hansen, Hanne E. H. Møller, Aleksandra Maria Rojek, Niels Marcussen, Hans Christian Beck, Niels Abildgaard   +5 more
doaj   +1 more source