Results 41 to 50 of about 35,531 (245)

Rationale, application and clinical qualification for NT-proBNP as a surrogate end point in pivotal clinical trials in patients with AL amyloidosis [PDF]

, 2016
Amyloid light-chain (LC) amyloidosis (AL amyloidosis) is a rare and fatal disease for which there are no approved therapies. In patients with AL amyloidosis, LC aggregates progressively accumulate in organs, resulting in organ failure that is ...
Ando, Y, Comenzo, RL, Dispenzieri, A, Gertz, MA, Grogan, M, Lousada, I, Maurer, MS, Merlini, G, Palladini, G, Sanchorawala, V, Wechalekar, A   +10 more
core   +1 more source

Cholesterol in mRNA‐Lipid Nanoparticles can be Replaced with the Synthetic Mycobacterial Monomycoloyl Glycerol Analogue MMG‐1

Advanced Functional Materials, EarlyView.
This study demonstrates that cholesterol in messenger RNA‐lipid nanoparticles (mRNA‐LNPs) can be completely replaced with an immunopotentiating lipid, i.e., a synthetic analogue of the C‐type lectin receptor agonist monomycoloyl glycerol (MMG‐1), without compromising physicochemical properties, in vivo transfection efficiency, and immunogenicity of the
Abhijeet G. Lokras, Saahil S. Baghel, Rune F. Jensen, Aneesh Thakur, Henrik Franzyk, Oskar Thofte, Marco Herrera‐Barrera, Madeleine Landry, Melike Ongun, Ahmad Tami, Marlene P. Thanthrige, Eva Callens, Corinne Beinat, Thomas Rades, Søren R. Paludan, Kristian Riesbeck, Ida Rosenkrands, Dennis Christensen, Gabriel K. Pedersen, Camilla Foged   +19 more
wiley   +1 more source

Cardiac Amyloidosis: Clinical Features, Pathogenesis, Diagnosis, and Treatment [PDF]

Türk Patoloji Dergisi
Cardiac amyloidosis is a type of amyloidosis that deserves special attention as organ involvement significantly worsens the prognosis. Cardiac amyloidosis can be grouped under three main headings: immunoglobulin light chain (AL) amyloidosis that is ...
Asuman ARGON, Deniz NART, Funda YILMAZ BARBET   +2 more
doaj   +1 more source

Monoclonal gammopathy of renal significance: Diagnostic workup [PDF]

, 2017
The clinical spectrum of diseases associated with monoclonal gammopathies is wide and they are most commonly the consequence of renal deposition of monoclonal immunoglobulin or its components.
Cabrita, A., Correia, S., Malheiro, J., Martins, L., Santos, J., Santos, S.   +5 more
core   +1 more source

High‐Throughput Strategies for Streamlining Lipid Nanoparticle Development Pipeline

Advanced Science, EarlyView.
This review highlights emerging state‐of‐the‐art high‐throughput strategies for optimising lipid nanoparticle formulation. By integrating combinatorial design, characterization, in vitro/in vivo screening, automation, and machine learning into a closed‐loop framework, it provides a roadmap to streamline discovery and accelerate the translation of ...
Lois Lam, Stephanie Watson, Yogambha Ramaswamy, Gurvinder Singh   +3 more
wiley   +1 more source

The impact of limited healthcare access among patients with light chain and transthyretin amyloidosis: real-world survey during COVID-19 lockdown period in France

Orphanet Journal of Rare Diseases
Background The containment strategies during the COVID-19 pandemic between December 2019 and 2022 significantly disrupted the healthcare system. Cardiac amyloidosis has a poor prognosis and requires frequent follow-up in reference centres.
D. Guijarro, A. Jobbe-Duval, S. Aguilhon, F. Bauer, E. Donal, J. C. Eicher, J. Jeanneteau, B. Gellen, D. Kenizou, O. Lairez, B. Lequeux, D. Legallois, P. Réant, M. Salvat, M. F. Seronde, M. Kharoubi, A. Whereat, A. Farrugia, C. Taieb, A. Zaroui, T. Damy   +20 more
doaj   +1 more source

Misdiagnosis of hereditary amyloidosis as AL (Primary) amyloidosis [PDF]

, 2002
Background: Hereditary, autosomal dominant amyloidosis, caused by mutations in the genes encoding transthyretin, fibrinogen A -chain, lysozyme, or apolipoprotein A-I, is thought to be extremely rare and is not routinely included in the differential ...
Booth, D.R., Booth, S.E., Bybee, A., Gilbertson, J.A., Gillmore, J.D., Hawkins, P.N., Lachmann, H.J., Pepys, M.B.   +7 more
core  

Nanoscopic Mapping of the Extracellular Space in Amyloid Plaque‐rich Cortex

Advanced Science, EarlyView.
The extracellular space and diffusion around amyloid plaques are examined using shadow imaging and single‐particle tracking. Increased diffusivity is found near plaques, extracellular matrix alterations, and plaque core penetrability that varies with amyloid phenotype.
Juan Estaún‐Panzano, Yulia Dembitskaya, Ivo Calaresu, Somen Nandi, Quentin Gresil, Evelyne Doudnikoff, Thierry Leste‐Lasserre, Thierry Amédée, Laurent Cognet, Laurent Groc, Urs Valentin Nägerl, Erwan Bezard   +11 more
wiley   +1 more source

Differences in immunoglobulin light chain species found in urinary exosomes in light chain amyloidosis (Al). [PDF]

PLoS ONE, 2012
Renal involvement is a frequent consequence of plasma cell dyscrasias. The most common entities are light chain amyloidosis, monoclonal immunoglobulin deposition disease and myeloma cast nephropathy.
Marina Ramirez-Alvarado, Christopher J Ward, Bing Q Huang, Xun Gong, Marie C Hogan, Benjamin J Madden, M Cristine Charlesworth, Nelson Leung   +7 more
doaj   +1 more source

IgM-Related Immunoglobulin Light Chain (AL) Amyloidosis

Hemato, 2022
Waldenström macroglobulinemia (WM) is a rare lymphoplasmacytic disorder characterized by an IgM paraprotein. The clinical presentation of WM varies and can include common manifestations such as anemia and hyperviscosity, in addition to less common ...
Shayna Sarosiek, Andrew R. Branagan, Steven P. Treon, Jorge J. Castillo   +3 more
doaj   +1 more source