tofersen (Qalsody)
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Indications
- treatment of amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene
Dosage
- intrathecal administration
- 100 mg (15 mL) per administration
- initiate with 3 loading doses administered at 14 day intervals
- maintenance dose every 28 days
Adverse effects
- > 10%
- fatigue, arthralgia, myalgia, increased CSF leukocyte count
* serious events of myelitis & radiculitis
* papilledema & increased intracranial pressure
Mechanism of action
- antisense oligonucleotide that targets mutated SOD1 genes to prevent expression of toxic SOD1 proteins
More general terms
References
- ↑ ALS Association BIOGEN IN PARTNERSHIP WITH IONIS PHARMACEUTICALS Tofersen https://www.als.org/navigating-als/living-with-als/fda-approved-drugs/tofersen
- ↑ HIGHLIGHTS OF PRESCRIBING INFORMATION QALSODY (tofersen) injection, for intrathecal use. https://www.biogencdn.com/us/pdfs/qalsody-prescribing-information.pdf