Article
Real-life experience with inotersen in hereditary transthyretin amyloidosis with late-onset phenotype: Data from an early-access program in Italy.
European journal of neurology - 1 Jul 2022
Luigetti Marco, Antonini Giovanni, Di Paolantonio Andrea, Gentile Luca, Grandis Marina, Leonardi Luca, Lozza Alessandro, Manganelli Fiore, Mazzeo Anna, Mussinelli Roberta, My Filomena, Obici Laura, Maria Pennisi Elena, Romozzi Marina, Russo Massimo, Sabatelli Mario, Salvalaggio Alessandro, Tagliapietra Matteo, Tozza Stefano
Abstract excerpt
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...
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