User:Mr. Ibrahem/Tofersen

Tofersen, sold under the brand name Qalsody, is a medication used to treat amyotrophic lateral sclerosis (ALS). Specifically it is used in people with a mutation of the gene superoxide dismutase 1 (SOD1), which is present in 2% of cases. It is given by injection into the spinal cord.

Common side effects include tiredness, joint pain, increased CSF white blood cells, and muscle pain. Other side effect may include myelitis, increased intracranial pressure, and aseptic meningitis. It is an antisense oligonucleotide which decreases superoxide dismutase 1 production.

Tofersen was approved for medical use in the United States in 2023. As of 2023 it costs about 158,000 USD per year in the United States. It received orphan medication status in Europe in 2016.