Results 81 to 90 of about 32,506 (196)
ESC Heart Failure, Volume 12, Issue 2, Page 727-729, April 2025.
Wojciech Kosmala +1 more
wiley +1 more source
Clinical Pharmacology of Eplontersen, the First Approved GalNAc‐Conjugated Antisense Oligonucleotide
Abstract Eplontersen is a GalNAc‐conjugated antisense oligonucleotide (ASO) approved for the treatment of hereditary transthyretin‐mediated amyloidosis with polyneuropathy (ATTRv‐PN). GalNAc conjugation enables targeted hepatic delivery and improved potency compared with unconjugated ASOs.
Rosie Z. Yu +4 more
wiley +1 more source
Early‐Onset Wild‐Type Transthyretin Amyloidosis Polyneuropathy
Muscle &Nerve, EarlyView.
Chafic Karam +7 more
wiley +1 more source
Amyotrophic lateral sclerosis (ALS) is a rare neurodegenerative disease for which there is currently no known cure. This systematic review explores the efficacy of genetic therapies used to target ALS in preclinical studies using in vivo rodent models. Outcomes investigated include the total number of motor neurons, rodent survival, and muscle function
Hannah E. Wedgwood +2 more
wiley +1 more source
Systemic ATTR-amyloidosis, a Rare Form of Internal Organ Damage [PDF]
The article presents the case report of a rare hereditary form of systemic ATTR-amyloidosis in Russian patient with a discussion of approaches to the diagnosis and treatment of this form, also based on the own experience in the management of such ...
S. V. Moiseev +12 more
core +1 more source
This systematic review and meta‐analysis of 13 observational studies found that catheter ablation was associated with lower odds of all‐cause mortality compared with medical management in cardiac amyloidosis and atrial fibrillation. Freedom from atrial arrhythmia after ablation was approximately 50%, with no significant associations for heart failure ...
Adivitch Sripusanapan +9 more
wiley +1 more source
Natural history and outcome in systemic AA amyloidosis [PDF]
BACKGROUND:Deposition of amyloid fibrils derived from circulating acute-phase reactant serum amyloid A protein (SAA) causes systemic AA amyloidosis, a serious complication of many chronic inflammatory disorders.
Goodman, HJB +6 more
core
Guidelines for genetic counselling in ATTR amyloidosis [PDF]
Genetic counselling is “a process of communication that deals with the occurrence, or risk of occurrence, of a (possibly) genetic disorder in the family” (ASHG, 1975; EuroGentest guidelines). It involves an attempt, by appropriately trained persons, to help patients and their families to (1) understand the medical facts of the disease; (2) appreciate ...
openaire +1 more source
BACKGROUND: Reduced LV longitudinal strain (GLS) and increased relative apical sparing (RELAPS) and increased wall thickness have been proposed as features for transthyretin cardiac amyloidosis (ATTR-CA).
Henein, Michael Y. +5 more
core +1 more source
Cardiac amyloidosis (CA) is a rare disorder caused by the deposition of abnormal proteins called amyloid in the myocardium, leading to dysfunction. The 2 most common forms of amyloidosis are AL (light chain) and ATTR (transthyretin).
Jose Loayza Pintado MD +3 more
doaj +1 more source

