Results 71 to 80 of about 71,732 (247)

Needs of amyloidosis patients and their care providers: design & first results of the  AMY-NEEDS research and care program

Orphanet Journal of Rare Diseases
Background Amyloidosis represents a rare yet heterogeneous multi-system disorder associated with a grave prognosis and an enormous psycho-emotional strain on patients, relatives, and caregivers.
Sandra Michaela Ihne-Schubert, Teresa Radovic, Saskia Fries, Stefan Frantz, Hermann Einsele, Stefan Störk, Silke Neuderth   +6 more
doaj   +1 more source

Dual-Energy SPECT and the Development of Peptide p5+14 for Imaging Amyloidosis

Molecular Imaging, 2017
Amyloidosis is associated with a number of rare diseases and is characterized by the deposition, in abdominothoracic organs and peripheral nerves, of extracellular protein fibrils, which leads to dysfunction and severe morbidity.
Jonathan S. Wall PhD, Stephen J. Kennel PhD, Emily B. Martin PhD   +2 more
doaj   +1 more source

Amyloidosis [PDF]

BMJ, 2011
Malvyn, Benjamin, Simon, Gibbs
openaire   +2 more sources

Physical stability of RNA–lipid nanoparticles under agitation, freeze–thaw and accelerated stability testing

Journal of Chemical Technology &Biotechnology, EarlyView.
Abstract BACKGROUND The choice of excipient is important in enhancing stability of liquid formulations containing lipid nanoparticles (LNPs) used in RNA delivery where the ability to survive unplanned excursions to higher and lower temperature and vibration is an important attribute.
Franz W Laurett Veras, Leah Wright, Robert J Falconer   +2 more
wiley   +1 more source

A Refractory Leg Skin Ulcer Associated With Multiple Myeloma Successfully Treated With Plasma Exchange, Lenalidomide, and Dexamethasone

JEADV Clinical Practice, EarlyView.
ABSTRACT Multiple myeloma (MM) is a malignant plasma cell disorder that primarily presents with CRAB symptoms (calcium elevation, renal failure, anemia, and bone abnormalities). In rare cases, MM manifests with systemic complications like skin ulcers, which present management challenges. Here, we report a 78‐year‐old Japanese man with MM and refractory
Naoko Hattori, Yutaka Kuwatsuka, Tomoka Waseda, Daisuke Ehara, Koji Ando, Hiroyuki Murota   +5 more
wiley   +1 more source

Successful Treatment of Refractory Lichen Amyloidosus With Dupilumab

JEADV Clinical Practice, EarlyView.
ABSTRACT Lichen amyloidosis (LA) is a chronic, pruritic dermatosis with papillary dermal amyloid deposition. Standard therapies often fail, and most reports of dupilumab in LA involve patients with concomitant atopic dermatitis. We report a 32‐year‐old man with refractory, non‐atopic LA who had failed topical corticosteroids, tacrolimus, phototherapy ...
Sohrab Elahwiesy, Franziska Jochims, Manfred Kunz, Susann Forkel, Jan‐Christoph Simon, Benjamin Klein   +5 more
wiley   +1 more source

Multiomics Insights Into AL Amyloidosis

Medicine Bulletin, EarlyView.
ABSTRACT Light chain amyloidosis is a systemic or localized protein conformational disorder triggered by misfolded immunoglobulin light chains, leading to amyloid fibril deposition. The disease is characterized by multiorgan involvement and delayed diagnosis, contributing to poor prognosis and high mortality rates.
Zixuan Zhang, Ziru Huang, Haoxiang Tang, Xianrun Pan, Rong Wang, Dingding Zhang, Jian Huang   +6 more
wiley   +1 more source

Ethnicity in systemic AL amyloidosis may impact risk stratification

Haematologica
Not available.
Jahanzaib Khwaja, Sriram Ravichandran, Oliver Cohen, Darren Foard, Ana Martinez – Naharro, Lucia Venneri, Carol Whelan, Philip N. Hawkins, Julian Gillmore, Helen J. Lachmann, Shameem Mahmood, Marianna Fontana, Ashutosh Wechalekar   +12 more
doaj   +1 more source

Iron deficiency in heart failure: Epidemiology, diagnostic criteria and treatment modalities

ESC Heart Failure, Volume 12, Issue 2, Page 723-726, April 2025.
Stephan von Haehling
wiley   +1 more source

Prime editing in neuropsychiatric disorders: From mutation‐specific target selection to clinical translation

Neuroprotection, EarlyView.
Abstract Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)‐based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR‐associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)‐encoded template.
Tianshan Ji, Yuan Zhang, Jinyi Zhao, Yiping Lu, Chengkun Wang   +4 more
wiley   +1 more source