About tofersen
Antisense oligonucleotide
Used for
- Treats adults with SOD1-associated amyotrophic lateral sclerosis (ALS).
Usual adult dose
100 mg by intrathecal injection three times, 14 days apart, then every 28 days.
Common side effects
- Pain, fatigue, joint or muscle pain
- Increased white blood cells in cerebrospinal fluid
- Headache, fever, or back pain
- Spinal cord inflammation or increased pressure around the brain
Important warnings
- Requires administration into the spinal canal by trained clinicians.
- Serious neurologic events, including myelitis and papilledema, have occurred.
- Monitor for neurologic symptoms and increased pressure around the brain.
- May cause low platelet counts or kidney inflammation; monitoring may be needed.
Pregnancy
Human pregnancy data are insufficient to assess risk.
Breastfeeding
It is unknown whether tofersen passes into breast milk; effects on infants are unknown.
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 It may be incomplete or out of date.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.