BIIB067 is an antisense oligonucleotide (ASO) inhibitor. It is designed to target the SOD1 gene. A mutation, or change, to the SOD1 gene has been shown to cause some forms of ALS. When there is a mutation to the SOD1 gene, it can cause the production of harmful or toxic proteins. These proteins may lead to the destruction of motor neurons—the cells that allow muscle movement in the body—and cause ALS.Am I Eligible?
Researchers are studying the investigational therapy because they believe it may reduce the amount of harmful or toxic SOD1 proteins and slow the progression of the disease.