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Long-term efficacy and safety of inotersen for hereditary transthyretin amyloidosis: NEURO-TTR open-label extension 3-year update

BACKGROUND: Hereditary transthyretin amyloidosis (hATTR/ATTRv) results from the deposition of misfolded transthyretin (TTR) throughout the body, including peripheral nerves. Inotersen, an antisense oligonucleotide inhibitor of hepatic TTR production, demonstrated a favorable efficacy and safety prof...

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Detalles Bibliográficos
Autores principales: Brannagan, Thomas H., Coelho, Teresa, Wang, Annabel K., Polydefkis, Michael J., Dyck, Peter J., Berk, John L., Drachman, Brian, Gorevic, Peter, Whelan, Carol, Conceição, Isabel, Plante-Bordeneuve, Violaine, Merlini, Giampaolo, Obici, Laura, Plana, Josep Maria Campistol, Gamez, Josep, Kristen, Arnt V., Mazzeo, Anna, Gentile, Luca, Narayana, Arvind, Olugemo, Kemi, Aquino, Peter, Benson, Merrill D., Gertz, Morie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9618524/
https://www.ncbi.nlm.nih.gov/pubmed/35908242
http://dx.doi.org/10.1007/s00415-022-11276-8