About onasemnogene abeparvovec
Adeno-associated viral gene therapy
Used for
- Treats spinal muscular atrophy in children with mutations in the SMN1 gene.
Usual adult dose
Not used in adults; treatment is for pediatric patients.
Usual dose in children
One intravenous infusion, dosed by body weight: 1.1 × 10¹⁴ vector genomes per kilogram.
Common side effects
- Elevated liver enzymes or liver injury
- Vomiting
- Thrombocytopenia
- Elevated troponin
Important warnings
- Can cause serious liver injury, including acute liver failure.
- Corticosteroids and frequent liver tests are required around treatment.
- Infusion reactions and low platelet counts can occur.
- Assess for pre-existing immunity to the vector before treatment.
Pregnancy
Not indicated for pregnancy; pregnancy-related safety information is limited.
Breastfeeding
Not indicated during breastfeeding; safety information is limited.
Interactions
- Vaccines may need to be timed around corticosteroid treatment; discuss vaccination plans with the treatment 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 It may be incomplete or out of date.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.