Results 31 to 40 of about 200,281 (336)

Inhibition by small-molecule ligands of formation of amyloid fibrils of an immunoglobulin light chain variable domain. [PDF]

, 2015
Overproduction of immunoglobulin light chains leads to systemic amyloidosis, a lethal disease characterized by the formation of amyloid fibrils in patients' tissues. Excess light chains are in equilibrium between dimers and less stable monomers which can
Brumshtein, Boris, Cascio, Duilio, Eisenberg, David S, Esswein, Shannon R, Ly, Alan T, Phillips, Martin L, Salwinski, Lukasz, Sawaya, Michael R   +7 more
core   +1 more source

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

New England Journal of Medicine, 2018
BACKGROUND Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin. METHODS In this phase 3 trial, we randomly assigned patients with hereditary transthyretin amyloidosis with ...
D. Adams, A. González-Duarte, W. O'Riordan, Chih-Chao Yang, M. Ueda, A. Kristen, I. Tournev, H. Schmidt, T. Coelho, J. Berk, Kon-Ping Lin, G. Vita, S. Attarian, V. Planté-Bordeneuve, M. Mezei, J. Campistol, J. Buades, T. Brannagan, Byoung-Joon Kim, Jeeyoung Oh, Y. Parman, Y. Sekijima, P. Hawkins, S. Solomon, M. Polydefkis, P. Dyck, P. Gandhi, S. Goyal, Jihong Chen, A. Strahs, S. Nochur, M. Sweetser, P. Garg, A. Vaishnaw, J. Gollob, O. Suhr   +35 more
semanticscholar   +1 more source

Global epidemiology of amyloid light-chain amyloidosis

Orphanet Journal of Rare Diseases, 2022
Background Amyloid light-chain (AL) amyloidosis is an ultra-rare disease associated with significant morbidity and mortality. Few studies have examined the global epidemiology of this condition.
Nishant Kumar, Nicole Zhang, D. Cherepanov, D. Romanus, M. Hughes, D. Faller   +5 more
semanticscholar   +1 more source

Clinical Amyloid Typing by Proteomics: Performance Evaluation and Data Sharing between Two Centres

Molecules, 2021
Amyloidosis is a relatively rare human disease caused by the deposition of abnormal protein fibres in the extracellular space of various tissues, impairing their normal function. Proteomic analysis of patients’ biopsies, developed by Dogan and colleagues
Diana Canetti, Francesca Brambilla, Nigel B. Rendell, Paola Nocerino, Janet A. Gilbertson, Dario Di Silvestre, Andrea Bergamaschi, Francesca Lavatelli, Giampaolo Merlini, Julian D. Gillmore, Vittorio Bellotti, Pierluigi Mauri, Graham W. Taylor   +12 more
doaj   +1 more source

Guidelines for non-transplant chemotherapy for treatment of systemic AL amyloidosis: EHA-ISA working group

Amyloid: Journal of Protein Folding Disorders, 2022
Background This guideline has been developed jointly by the European Society of Haematology and International Society of Amyloidosis recommending non-transplant chemotherapy treatment for patients with AL amyloidosis.
A. Wechalekar, M. Cibeira, S. Gibbs, A. Jaccard, Shaji K. Kumar, G. Merlini, G. Palladini, V. Sanchorawala, S. Schönland, C. Venner, M. Boccadoro, E. Kastritis   +11 more
semanticscholar   +1 more source

Systemic amyloidosis in England: an epidemiological study. [PDF]

, 2013
Epidemiological studies of systemic amyloidosis are scarce and the burden of disease in England has not previously been estimated. In 1999, the National Health Service commissioned the National Amyloidosis Centre (NAC) to provide a national clinical ...
Banypersad, SM, Dungu, J, Gibbs, SD, Gillmore, JD, Hawkins, PN, Lachmann, HJ, Pinney, JH, Smith, CJ, Taube, JB, Venner, CP, Wechalekar, AD, Whelan, CJ   +11 more
core   +1 more source

Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update

Orphanet Journal of Rare Diseases, 2022
Background Transthyretin amyloidosis (ATTR amyloidosis) is a rare, life-threatening disease caused by the accumulation of variant or wild-type (ATTRwt amyloidosis) transthyretin amyloid fibrils in the heart, peripheral nerves, and other tissues and ...
A. Dispenzieri, T. Coelho, I. Conceição, M. Waddington-Cruz, J. Wixner, A. Kristen, C. Rapezzi, V. Planté-Bordeneuve, J. González-Moreno, M. Maurer, M. Grogan, Doug Chapman, L. Amass, Pablo Garcia Ivaylo Jose Gonzalez Maria Alejandra Gonzal Pavia Tarnev Costello Briseno Schmidt Drachman Bar, P. G. Pavía, Ivaylo Tarnev, J. Costello, Maria Alejandra González-Duarte Briseño, H. Schmidt, B. Drachman, F. Barroso, T. Yamashita, O. Lairez, Y. Sekijima, G. Vita, E. Jeon, M. Hanna, D. Slosky, M. Luigetti, S. Lorusso, F. Beamud, D. Adams, H. Moelgaard, R. Press, C. Cirami, H. Nienhuis, J. M. Plana, J. Inamo, D. Jacoby, M. Emdin, D. Quan, S. Hummel, R. Witteles, A. Dori, Sanjiv Shah, D. Lenihan, O. Azevedo, S. Murali, S. Živković, S. Low, J. Nativi-Nicolau, N. Fine, J. Tallaj, C. Tschoepe, R. Torrón, M. Polydefkis, G. Merlini, S. Bădeliță, Stephen S. Gottlieb, J. Tauras, E. B. Correia, H. Ventura, B. Gess, F. Darstein, Jeeyoung Oh, Tessa Marburger, J. Van Cleemput, V. Salutto, Y. Parman, C. Chao, N. Sarswat, Christopher Mueller, D. Steidley, J. Ralph, A. Warner, W. Cotts, J. Hoffman, M. Rugiero, S. Misawa, J. L. Blanco, L. G. Dávila, M. Sadeh, Jinju Luo, T. Kyriakides, Annabel K. Wang, H. Kaufmann, S. Živković   +86 more
semanticscholar   +1 more source

Atrial amyloidosis: mechanisms and clinical manifestations

European Journal of Heart Failure, 2022
Cardiac amyloidosis (CA) is now recognized as an important cause of heart failure. Increased wall thickness and diastolic dysfunction of the left ventricle are the most easily detectable manifestations of CA, but amyloid accumulates in all cardiac ...
G. Vergaro, A. Aimo, C. Rapezzi, V. Castiglione, I. Fabiani, A. Pucci, G. Buda, C. Passino, J. Lupón, A. Bayés‐Genís, M. Emdin, E. Braunwald   +11 more
semanticscholar   +1 more source

Human central nervous system (CNS) ApoE isoforms are increased by age, differentially altered by amyloidosis, and relative amounts reversed in the CNS compared with plasma [PDF]

, 2016
The risk of Alzheimer's disease (AD) is highly dependent on apolipoprotein-E (apoE) genotype. The reasons for apoE isoform-selective risk are uncertain; however, both the amounts and structure of human apoE isoforms have been hypothesized to lead to ...
Baker-Nigh, Alaina T, Bateman, Randall J, Bollinger, James G, Cairns, Nigel J, Franklin, Erin E, Holtzman, David, Jiang, Hong, Kasten, Tom, Liao, Fan, Mawuenyega, Kwasi G, Morris, John C, Ovod, Vitaliy   +11 more
core   +3 more sources

Comparative Outcomes of a Transthyretin Amyloid Cardiomyopathy Cohort Versus Patients With Heart Failure With Preserved Ejection Fraction Enrolled in the TOPCAT Trial

Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease, 2023
Background Transthyretin cardiac amyloidosis (ATTR‐CM), found in 6% to 15% of cohorts with heart failure with preserved ejection fraction, has long been considered a rare disease with poor prognosis.
Morris M. Kim, Mark Prasad, Yunwoo Burton, Clinton M. Kolseth, Yuanzi Zhao, Pranav Chandrashekar, Babak Nazer, Ahmad Masri   +7 more
doaj   +1 more source