Auflistung Enfermedades Neuromusculares.pósters nach Autor "Berk, John L."

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Eplontersen in Hereditary ATTR-polyneuropathy: Week 66 Final Analysis of the Phase 3 NEURO-TTRansform Study

Khella, Sami L.; Marques, Wilson Jr.; Chao, Chi-Chao; Yesim Parman, Fatma; Franca, Marcondes C.; Guo, Yuh-Cherng; Ro, Long-Sun; Calandra, Cristian; Kowacs, Pedro A.; Berk, John L.; Piera Obici, Laura; Barroso, Fabio Adrián; Conceicao, Isabel; Schneider, Eugene; Viney, Nicholas; Dyck, James B.; Waddington Cruz, Marcia; Coelho, Teresa (American Academy of Neurology, 2023-04-24)

Background ATTRv-PN is a rare, progressive, and debilitating disease caused by accumulation of amyloid fibrils composed of transthyretin (TTR) protein in multiple organ systems. Eplontersen, a ligand-conjugated antisense ...
Long-Term Efficacy and Safety of Inotersen for Hereditary Transthyretin Amyloidosis: NEURO-TTR Open-Label Extension 2-Year Update (S27.008)

Brannagan, Thomas H.; Waddington Cruz, Marcia; Wang, Annabel K.; Polydefkis, Michael J.; Dyck, Peter J.; Khella, Sami; Plante-Bordeneuve, Violaine; Berk, John L.; Barroso, Fabio Adrián; Merlini, Giampaolo; Conceição, Isabel; Hughes, Steven G.; Kwoh, Jesse; Jung, Shiangtung W.; Guthrie, Spencer; Pollock, Michael; Benson, Merrill D.; Gertz, Morie; Coelho, Teresa (Lippincott Williams & Wilkins, 2019-03-09)

Objective: To provide an update on the long-term efficacy and safety of inotersen, an antisense oligonucleotide inhibitor of transthyretin protein production, in patients with hereditary transthyretin amyloidosis (hATTR) ...

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