Real-life experience with inotersen in hereditary transthyretin amyloidosis with late-onset phenotype: Data from an early-access program in Italy.

ABSTRACT:

Background and purpose

Hereditary transthyretin (TTR) amyloidosis (ATTRv) is a dominantly inherited, adult-onset, progressive, and fatal disease caused by mutations in the transthyretin gene. Therapeutic agents approved for this disease include the TTR stabilizer tafamidis and the gene-silencing drugs patisiran and inotersen. Inotersen is an antisense oligonucleotide that suppresses the hepatic production of transthyretin. After European Medical Agency approval in 2018, an early-access program was opened in Italy, and in this article, we present the long-term outcome of a cohort of Italian ATTRv patients who received inotersen within this program.

Methods

This is a multicenter, observational, retrospective study of patients affected by ATTRv that started inotersen during the

SUBMITTER: Luigetti M 

PROVIDER: S-EPMC9314115 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature