The findings of two recent studies give hope that the disease could one day be reversed in humans—but experts warn that this ...

Scientists at the University of Sydney have uncovered a malfunctioning version of the SOD1 protein that clumps inside brain ...

A team of neuroscientists in Australia has found that restoring copper levels in the brain dramatically reduced Parkinson-like damage in mice. The approach restored a protein’s function, and the same ...

Groundbreaking research by Sydney University found a new brain protein involved in Parkinson’s disease and a way to modify it ...

Groundbreaking research by the University of Sydney has identified a new brain protein involved in the development of Parkinson's disease and a way to modify it, paving the way for future treatments ...

A newly discovered brain protein linked to Parkinson’s offers hope for targeted therapies that could slow or prevent disease progression in the future.

The disease, which is caused by mutations in the SOD1 gene, accounts for approximately 10% to 20% of genetic ALS cases and 1% to 2% of sporadic ALS cases, according to the ALS Association. 7.

Tofersen (BIIB067) is an ASO that is under investigation for the treatment of ALS caused by SOD1 mutations. Tofersen has been designed to mediate RNase H–dependent degradation of SOD1 mRNA to ...

The U.S. Food and Drug Administration approved a new drug Tuesday to treat a rare form of amyotrophic lateral sclerosis, or ALS.. The drug Qalsody (tofersen) is expected to help people with a very ...

In this phase 3 trial, we randomly assigned adults with SOD1 ALS in a 2:1 ratio to receive eight doses of tofersen (100 mg) or placebo over a period of 24 weeks. The primary end point was the ...