Extended Data Table 4 AEs of special interest

From: AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial

  1. aGGT or GLDH > 3× ULN and the new appearance (that is, onset coincides with the changes in hepatic enzymes) of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash or eosinophilia (>5%) potentially related to hepatic inflammation.
  2. bTroponin I > 3× ULN for patients with non-elevated baseline values (baseline troponin I > ULN) or troponin I > 3× baseline for patients with elevated baseline values (baseline troponin I > ULN).
  3. bGGT, gamma-glutamyl transferase; ULN, upper limit of normal.
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