Browsing by Author "Conceicao, I."
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- Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose studyPublication . Suhr, O.; Coelho, T.; Buades, J.; Pouget, J.; Conceicao, I.; Berk, J.; Schmidt, H.; Waddington-Cruz, M.; Campistol, J.; Bettencourt, B.; Vaishnaw, A.; Gollob, J.; Adams, D.BACKGROUND: Transthyretin-mediated amyloidosis is an inherited, progressively debilitating disease caused by mutations in the transthyretin gene. This study evaluated the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses of patisiran (ALN-TTR02), a small interfering RNA encapsulated within lipid nanoparticles, in patients with transthyretin-mediated familial amyloid polyneuropathy (FAP).