Table 3 Summary of adverse events (AEs) in patients with SMA receiving onasemnogene abeparvovec early in life.
From: Early life safety profiling of gene therapy for spinal muscular atrophy
| Â | Mild/Moderate | Severe/Hospital visit needed | Total | % |
|---|---|---|---|---|
Fever | 8 | 1 | 9 | 11.4% |
GI | 9 | --- | 9 | 11.4% |
Cardiac/Acute life threatening | 1 | 1 | 2 | 2.5% |
Respiratory | 6 | 3 | 9 | 11.4% |
Orthopedic | 5 | --- | 5 | 6.3% |
Nutritional | 1 | 1 | 2 | 2.5% |
Other | 39 | 4 | 43 | 54.4% |
TOTAL AEs | 69 | 7 | 79 | Â |
% | 87.3% | 8.9% | Â | Â |
