Table 2.
Summary of safety.
| AE, n (%) | 2 SMN2 copies n = 15 | 3 SMN2 copies n = 10 | Total N = 25 |
|---|---|---|---|
| Any AE | 15 (100) | 10 (100) | 25 (100) |
| Serious AE | 9 (60) | 3 (30) | 12 (48) |
| Severe AE | 5 (33) | 0 | 5 (20) |
| AE related to study druga | 0 | 0 | 0 |
| AE possibly related to study druga | 4 (27) | 4 (40) | 8 (32) |
| Proteinuria | 1 (7) | 1 (10) | 2 (8) |
| Increased alkaline phosphatase and calcium | 1 (7) | 0 | 1 (4) |
| Muscular weakness, weight-bearing difficulty, extensor plantar response, clonus | 1 (7) | 0 | 1 (4) |
| Hyperreflexia and tachycardia | 0 | 1 (10) | 1 (4) |
| Pyrexia, increased ALT, increased AST, increased eosinophil, lymphocyte, and WBC counts | 1 (7) | 0 | 1 (4) |
| Increased platelet count | 0 | 1 (10) | 1 (4) |
| Rash | 0 | 1 (10) | 1 (4) |
| Serious AE related to study druga | 0 | 0 | 0 |
| AE possibly related to lumbar puncture procedureb | 6 (40) | 2 (20) | 8 (32) |
| AE leading to treatment discontinuation or study withdrawal | 0 | 0 | 0 |
| Incidence of AEs by MedDRA PT occurring in ≥5 participants | |||
| Pyrexia | 14 (93) | 7 (70) | 21 (84) |
| Upper respiratory tract infection | 12 (80) | 7 (70) | 19 (76) |
| Cough | 8 (53) | 5 (50) | 13 (52) |
| Nasopharyngitis | 9 (60) | 4 (40) | 13 (52) |
| Vomiting | 7 (47) | 3 (30) | 10 (40) |
| Fall | 4 (27) | 5 (50) | 9 (36) |
| Rhinorrhea | 5 (33) | 4 (40) | 9 (36) |
| Nasal congestion | 5 (33) | 3 (30) | 8 (32) |
| Otitis media | 4 (27) | 4 (40) | 8 (32) |
| Diarrhea | 2 (13) | 5 (50) | 7 (28) |
| Influenza | 4 (27) | 3 (30) | 7 (28) |
| Seasonal allergy | 3 (20) | 4 (40) | 7 (28) |
| Tremor | 7 (47) | 0 | 7 (28) |
| Anemia | 3 (20) | 3 (30) | 6 (24) |
| Constipation | 5 (33) | 1 (10) | 6 (24) |
| Dehydration | 5 (33) | 1 (10) | 6 (24) |
| Pneumonia | 6 (40) | 0 | 6 (24) |
| Diaper dermatitis | 3 (20) | 2 (20) | 5 (20) |
| Viral gastroenteritis | 3 (20) | 2 (20) | 5 (20) |
| Muscular weakness | 5 (33) | 0 | 5 (20) |
| Tachycardia | 4 (27) | 1 (10) | 5 (20) |
AE, adverse event; ALT, alanine aminotransferase, AST, aspartate aminotransferase; MedDRA PT, Medical Dictionary for Regulatory Activities Preferred Term; SMN2, survival motor neuron 2.
Values refer to numbers and percentages of study participants.
a
Assessed by the investigator.
b
Assessed by the investigator; includes events that are possibly related or related to the LP procedure.