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Long-term efficacy and safety of inotersen for hereditary transthyretin amyloid...

Brannagan, Thomas H.; Coelho, Teresa; Wang, Annabel K.; Polydefkis, Michael J.; Dyck, Peter J.; Berk, John L.; Drachman, Brian; Gorevic, Peter

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 profile in patients with the polyneuropathy associated with hATTR in the NEURO-TTR (NCT01737398) study. We report long...

Date: 2022   |   Origin: Repositório da Universidade de Lisboa
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