Tofersen sodium

Unassigned

New Medicines

Amyotrophic lateral sclerosis (motor neurone disease)

Information

New molecular entity
Biogen
Biogen

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes

Category

An antisense therapy, BIIB067 suppresses production of a mutant protein, Cu/Zn superoxide dismutase (SOD1), associated with an aggressive form of the disease. Reduction of SOD1 protein in the brain may modify or halt the progression of ALS.
MND is relatively uncommon with an annual incidence of about 2 cases per 100,000 population. Prevalence is about 5-7 per 100,000. General practitioners can expect to see one or two cases in their career [1].
Amyotrophic lateral sclerosis (motor neurone disease)
Intrathecal