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Fig. 1 | Orphanet Journal of Rare Diseases

Fig. 1

From: Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

Fig. 1

Dose response and duration of TTR knockdown. Mean (±SEM) percentage of baseline serum concentration–time profile. Q3W: every 3 weeks; Q4W: every 4 weeks; SEM: standard error of the mean; TTR: transthyretin. *Includes first dose data from additional patient prior to protocol amendment. Excludes post-Day 28 data from patient who experienced drug extravasation during second infusion. One patient discontinued before the second dose of patisiran

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