Browsing Enfermedades Neuromusculares.pósters by Subject "Neuropatías Amiloides Familiares"

Now showing items 1-2 of 2

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

Khella, Sami; Marques, Wilson; 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; Conceição, Isabel; Schneider, Eugene; Viney, Nicholas J.; Dyck, P. 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 ...
Familial Amyloid Polyneuropathy: Impact of Biopsies and Mutations on Diagnostic Considerations (P3.4-033)

Gibbons, Christopher; González-Duarte, Alejandra; Barroso, Fabio Adrián; Campagnolo, Marta; Rajan, Sharika; Freeman, Roy (AAN, 2019-05-07)

Objective: To characterize the symptoms, signs and skin biopsy neuropathological findings in a cohort of individuals with TTR Mutations. Background: Familial amyloid polyneuropathy is due to one of many mutations in the ...

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