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

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 dr...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Neuropathies
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9314115/
https://www.ncbi.nlm.nih.gov/pubmed/35289020
http://dx.doi.org/10.1111/ene.15325

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9314115/
https://www.ncbi.nlm.nih.gov/pubmed/35289020
http://dx.doi.org/10.1111/ene.15325

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