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The overall mean 30-minute incremental recovery was 1.66 IU/dL per IU/kg for patients’ baseline visit, 1.82 IU/dL per IU/kg for the end-of-study visit, and 1.74 IU/dL per IU/kg for both visits combined.

Incremental recovery was significantly lower in the 0-5 age group than the 6-11 age group:

• 1.45 vs 1.83 at baseline (P=0.027)
• 1.62 vs 1.99 at end of study (P=0.025)
• 1.53 vs 1.91 combined (P=0.001).

All patients had FX:C trough levels greater than 5% after visit 4 (days 29-42).

There were 28 AEs reported in 8 patients, but none of these events were considered related to treatment.

One patient did have 2 serious treatment-emergent AEs—lower respiratory tract infection and influenza.

There were no other serious AEs, no evidence of factor X inhibitor development, and no deaths.