Qalsody

Qalsody is a medicine for treating adults with a type of amyotrophic lateral sclerosis (ALS) caused by a mutation (defect) in the gene responsible for producing an enzyme called superoxide dismutase 1 (SOD1). ALS is a progressive disease of the nervous system where nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing loss of muscle function and paralysis. ALS is rare, and Qalsody was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 29 August 2016. ALS caused by a mutation in the SOD1 gene represents about 2% of patients with ALS. Qalsody contains the active substance tofersen.