Results 1 to 10 of about 148,376 (307)

ASNC/AHA/ASE/EANM/HFSA/ISA/SCMR/SNMMI expert consensus recommendations for multimodality imaging in cardiac amyloidosis: Part 2 of 2—Diagnostic criteria and appropriate utilization [PDF]

Journal of Nuclear Cardiology, 2019
Sharmila Dorbala, Yukio Ando, Sabahat Bokhari, Angela Dispenzieri, Rodney H. Falk, Victor A. Ferrari, Marianna Fontana, Olivier Gheysens, Julian D. Gillmore, Andor W.J.M. Glaudemans, Mazen Hanna, Bouke P. C. Hazenberg, Arnt V. Kristen, Raymond Y. Kwong, Matthew J. Maurer, Giampaolo Merlini, Edward J. Miller, James Moon, Venkatesh L. Murthy, Cristina Quarta, Claudio Rapezzi, Frederick L. Ruberg, Sanjiv J. Shah, Riemer H. J. A. Slart, Hein J. Verberne, Jamieson M. Bourque   +25 more
openalex   +2 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

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

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, K. Tsujita, W. Tang, Wen-Chung Yu, L. Obici, M. Simões, Fábio Fernandes, S. Poulsen, Igor Diemberger, F. Perfetto, S. Solomon, Marcelo 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

Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy.

Journal of the American Medical Association (JAMA), 2023
Importance Transthyretin gene silencing is an emerging treatment strategy for hereditary transthyretin (ATTRv) amyloidosis. Objective To evaluate eplontersen, an investigational ligand-conjugated antisense oligonucleotide, in ATTRv polyneuropathy ...
Teresa Coelho, Wilson Marques, N. Dasgupta, C. Chao, Y. Parman, M. França, Yuh-Cherng Guo, J. Wixner, Long-Sun Ro, Cristian R Calandra, P. Kowacs, J. Berk, L. Obici, F. A. Barroso, M. Weiler, I. Conceição, Shiangtung W. Jung, G. Buchele, Michela Brambatti, Jersey Chen, Steven G. Hughes, Eugene Schneider, N. Viney, Ahmad Masri, Morie Gertz, Yukio Ando, J. Gillmore, S. Khella, P. J. B. Dyck, M. Waddington Cruz, Anna Mazzeo, A. Papagianni, Mazen Dimachkie, Ioannis Zaganas, Edward Gane, M. Luigetti, L. Galán Dávila, Michelle Mezei, Juan Gonzalez Moreno, P. Cintas, D. Pareyson, Rebecca Traub, Julie Khoury, C. Estol, Merrilee Needham, David John Adams, M. Polydefkis, T. Brannagan, V. Bril, S. Attarian, Marcelo Rugiero, Barbara Distad, Eleni Zamba Papanicolaou, Kon-Ping Lin, Merrill D. Benson, M. Scheinberg   +55 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

Impact of early response on outcomes in AL amyloidosis following treatment with frontline Bortezomib

Blood Cancer Journal, 2021
The outcomes in systemic AL amyloidosis are dependent on the depth of haematologic response. However, there is limited data on the impact of the speed of response on outcomes.
Sriram Ravichandran, Oliver C. Cohen, Steven Law, Darren Foard, Marianna Fontana, Ana Martinez-Naharro, Carol Whelan, Julian D. Gillmore, Helen J. Lachmann, Sajitha Sachchithanantham, Shameem Mahmood, Philip N. Hawkins, Ashutosh D. Wechalekar   +12 more
doaj   +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

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