Results 81 to 90 of about 8,194 (217)
Photo‐Catalyzed Labeling of Amyloid Deposits in Human Tissue to Proteotype Amyloidosis Diseases
Amyloid‐ID, a ThT‐like photocatalytic probe, enables proteotyping of amyloid deposits by ROS‐mediated protein labeling. Applied to patient biopsies, amyloid‐ID identifies potential fibrinogen‐derived amyloid in laryngeal amyloidosis, revealing a mechanically sensitive fibrillation mechanism linked to vocal cord friction.
Huan Feng +10 more
wiley +1 more source
Corino de Andrade disease: mechanisms and impact on reproduction [PDF]
Familial amyloid polyneuropathy was first described by Corino de Andrade in 1952 in Northern Portugal. It is a fatal autosomal dominant neurodegenerative disorder characterized by a progression of neurologic symptoms, beginning early in the reproductive ...
Barros, A. +3 more
core +1 more source
ABSTRACT Amyloidosis is a rare disease associated with the deposition of misfolded proteins that damage multiple organs, leading to a wide range of symptoms. The most frequently implicated proteins in amyloidosis include immunoglobulin Free Light Chains (FLC), related to AL amyloidosis, and transthyretin (TTR), which is responsible for ATTR amyloidosis.
Paulina Kryszpin +8 more
wiley +1 more source
ABSTRACT Background Hereditary transthyretin‐mediated amyloidosis (hATTR) is a disorder that affects several body systems and can result in life‐threatening conditions like cardiomyopathy and polyneuropathy. For treatment of hATTR in the new medical realm, there is a breakthrough discovery of a medicine called Eplontersen, which targets the underlying ...
Zeel Vishnubhai Patel +8 more
wiley +1 more source
Background Myeloproliferative disorders, including monoclonal gammopathy of undetermined significance (MGUS), are often associated with amyloid light-chain (AL)-type cardiac amyloidosis (CA) but occasionally with wild-type transthyretin (ATTR) CA.
Tomoaki Haga +8 more
doaj +1 more source
Progress and challenges in the treatment of cardiac amyloidosis: a review of the literature
Cardiac amyloidosis is a restrictive cardiomyopathy determined by the accumulation of amyloid, which is represented by misfolded protein fragments in the cardiac extracellular space.
Robert Daniel Adam +6 more
doaj +1 more source
Hereditary transthyretin amyloidosis: baseline characteristics of patients in the NEURO-TTR trial [PDF]
Background: Hereditary transthyretin (ATTRm) amyloidosis is a rare, progressive and fatal disease with a range of clinical manifestations.Objective: This study comprehensively evaluates disease characteristics in a large, diverse cohort of patients with ...
Ackermann, Elizabeth J. +16 more
core +3 more sources
RNA‐Based Therapies for Inherited Metabolic Disorders
ABSTRACT Inherited metabolic disorders (IMDs) are a diverse and complex group of genetic conditions resulting from deficiencies in enzymes, transporters, or cofactors. These deficiencies lead to metabolic dysfunction and severe clinical consequences. Despite significant progress in understanding their molecular basis, treatment options remain limited ...
Reddy Sreekanth Vootukuri +5 more
wiley +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
ABSTRACT Background and Aims In individuals with hereditary transthyretin amyloidosis (ATTRv) polyneuropathy, monitoring of disease progression and treatment response is crucial. The objective is to determine if serum neurofilament light chain (sNfL) and serum glial fibrillary acidic protein (sGFAP) are reliable biomarkers of ATTRv polyneuropathy ...
Valentin Loser +9 more
wiley +1 more source

