About onasemno gebe abeparvov
Adeno-associated virus gene therapy
Used for
- Treats spinal muscular atrophy caused by changes in the SMN1 gene in children under two years.
Usual adult dose
Not used in adults.
Usual dose in children
One intravenous infusion, dosed by body weight: 1.1 × 10¹⁴ vector genomes/kg.
Common side effects
- Raised liver enzymes or liver injury
- Vomiting
- Fever
- Low platelet count
Important warnings
- Can cause serious, potentially life-threatening liver injury.
- Corticosteroids are given before and after infusion; liver tests require monitoring.
- Serious infections may occur; delay treatment during active infection.
- May increase risk of thrombotic microangiopathy, a serious blood disorder.
Pregnancy
Not applicable to its intended use in children; pregnancy data are unavailable.
Breastfeeding
Not applicable to its intended use in children; effects during breastfeeding are unknown.
Interactions
- Live vaccines: avoid during corticosteroid treatment; timing should be reviewed by the treating team.
AI-generated from general medical knowledge, not from this product's official leaflet. It may be incomplete or out of date. Not medical advice — ask a pharmacist or doctor before making any decision.