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Table 2 Summary of adverse events

From: Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study

Event, n (%)

Overall (N = 9)

Any TEAE

9 (100)

Any related TEAE

6 (67)

Any IAR

5 (56)

Any serious TEAE

9 (100)

Any related serious TEAE

1 (11)

Dose modification due to a TEAE*

7 (78)

Discontinuation due to a TEAE

0

Death

4 (44)

Death related to treatment

0

  1. IAR infusion-associated reaction, TEAE treatment-emergent adverse event
  2. *All dose modifications were dose escalations based on inadequate clinical response. These symptoms of inadequate clinical response were also captured as adverse events (see Additional file 1)
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172