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. 2021 Sep 1;5(6):e12586. doi: 10.1002/rth2.12586

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© 2021 The Authors. Research and Practice in Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis (ISTH).

This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.

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Factor IX (FIX) activity across time (uniQure FIX clinical trial data). (A) Cohort 1; (B) Cohort 2. Only values at least 10 days after the preceding FIX concentrate administration, so that they are uncontaminated by exogenous FIX, are included. Participant 3 continued with prophylaxis after AMT‐060 treatment; as a result, only limited samples uncontaminated by exogenous FIX were available. The dotted line at FIX activity of 2 IU/dL indicates the threshold required for ceasing prophylaxis per protocol. FIX prophylaxis was continued after AMT‐060 and tapered between week 6 and week 12. Participant 4 had a moderate hemophilia B phenotype at baseline (FIX activity, 1.5 IU/dL). (Reproduced, with permission, from Miesbach et al, p 1027, Figure 2)