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Table 2 List of SAEs by MedDRA system organ class

From: Short-term safety results from compassionate use of risdiplam in patients with spinal muscular atrophy in Germany

Serious adverse events, n (%)*

SMA1

SMA2

Total

Total number

2 (6.7)

8 (8.0)

10 (7.7)

Gastrointestinal disorders

–

5 (5.0)

5 (3.8)

General disorders and administration site conditions

–

1 (1.0)

1 (0.8)

Infections and infestations

2 (6.7)

2 (2.0)

4 (3.0)

  1. Of 111 patients who received at least one dose of risdiplam (31 patients with SMA1 and 80 patients with SMA2), 3 patients (1 patient with SMA1 and 2 patients with SMA2) experienced at least 1 SAE
  2. *Percentages are based on total number of AEs (30 in SMA1 and 100 in SMA2). MedDRA, Medical Dictionary for Regulatory Activities; SAE, serious adverse event; SMA1, spinal muscular atrophy type 1; SMA2, spinal muscular atrophy type 2
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172