Specific Therapy in Transthyretin Amyloid Cardiomyopathy: Future Perspectives Beyond Tafamidis.
Heart Fail Clin
; 20(3): 343-352, 2024 Jul.
Article
in En
| MEDLINE
| ID: mdl-38844305
ABSTRACT
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a relatively prevalent cause of morbidity and mortality. Over the recent years, development of disease-modifying treatments has enabled stabilization of the circulating transthyretin tetramer and suppression of its hepatic production, resulting in a remarkable improvement in survival of patients with ATTR-CM. Second-generation drugs for silencing are currently under investigation in randomized clinical trials. In vivo gene editing of transthyretin has been achieving unanticipated suppression of hepatic production in ATTR-CM. Trials of antibodies inducing the active removal of transthyretin amyloid deposits in the heart are ongoing, and evidence has gathered for exceptional spontaneous regression of ATTR-CM.
Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Benzoxazoles
/
Prealbumin
/
Amyloid Neuropathies, Familial
/
Cardiomyopathies
Limits:
Humans
Language:
En
Journal:
Heart Fail Clin
Year:
2024
Document type:
Article