Results 11 to 20 of about 21,795 (202)
Therapeutic Efficacy and Safety Profile of Eplontersen in Hereditary Transthyretin-Mediated Amyloidosis: A Systematic Review. [PDF]
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 ...
Patel ZV +8 more
europepmc +2 more sources
Transthyretin amyloidosis [PDF]
Abstract: Cardiac amyloidosis is a poorly understood cause of heart failure and is often undiagnosed. Recent advances in diagnostic testing and understanding of the disease have enhanced the ability of clinicians to detect this disease and provide patients with appropriate treatment.
Renáta Aiglová +3 more
openaire +3 more sources
Transthyretin cardiac amyloidosis
AbstractTransthyretin cardiac amyloidosis (ATTR-CA) is an increasingly recognized cause of heart failure (HF) and mortality worldwide. Advances in non-invasive diagnosis, coupled with the development of effective treatments, have shifted ATTR-CA from a rare and untreatable disease to a relatively prevalent condition that clinicians should consider on a
Aldostefano Porcari +2 more
openaire +4 more sources
Negative [99mTc]Tc -DPD Scintigraphy, Presence of Monoclonal Protein and Biopsy Suggestive of AL Amyloidosis in a Patient With Homozygous p.Ala101Val Transthyretin Gene Variant. [PDF]
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.
Kryszpin P +8 more
europepmc +2 more sources
Abstract Background and Aims Intrahepatic cholangiocarcinoma (ICC) is a deadly but poorly understood disease, and its treatment options are very limited. The aim of this study was to identify the molecular drivers of ICC and search for therapeutic targets.
Yuto Shiode +16 more
wiley +1 more source
Transthyretin cardiac amyloidosis.
Transthyretin amyloid cardiomyopathy (ATTR-CM) may be an under recognized cause of heart failure (HF). TTR amyloidosis can be inherited, caused by variants in the TTR gene (ATTRv) or by deposition of wild-type TTR protein (ATTRwt), leading to high mortality if untreated.
Tomoaia, Raluca +6 more
openaire +2 more sources
Transthyretin amyloidosis (ATTR) is a progressive and systemic disease caused by the misfolding and amyloid aggregation of transthyretin (TTR). Stabilizing the TTR tetramers and disrupting the formed TTR aggregation are treated as a promising strategy for the treatment of ATTR.
Huizhen Zou, Shuangyan Zhou
openaire +2 more sources
BACKGROUND: Transthyretin amyloidosis (ATTR) is a rare, life-threatening systemic disorder. We present first findings on the cardiac hereditary ATTR in Poland.
Monika Gawor +11 more
doaj +1 more source
Hereditary cardiac amyloidosis associated with Pro24Ser transthyretin mutation: a case report
Background Transthyretin amyloidosis is a systemic disorder caused by extracellular deposition of insoluble amyloid fibrils in peripheral and autonomic nerves, heart, kidney, gastrointestinal tract, and other organs.
Hiroyuki Yamamoto +6 more
doaj +1 more source
Hereditary transthyretin amyloidosis overview [PDF]
AbstractHereditary amyloidogenic transthyretin (ATTRv) amyloidosis is a rare autosomal dominantly inherited disorder caused by mutations in the transthyretin (TTR) gene. The pathogenetic model of ATTRv amyloidosis indicates that amyloidogenic, usually missense, mutations destabilize the native TTR favouring the dissociation of the tetramer into ...
Manganelli, Fiore +5 more
openaire +5 more sources

