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Groundbreaking research by Sydney University found a new brain protein involved in Parkinson’s disease and a way to modify it ...
Scientists at the University of Sydney have uncovered a malfunctioning version of the SOD1 protein that clumps inside brain ...
Groundbreaking research by the University of Sydney has identified a new brain protein involved in the development of ...
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 ...
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.
The decision on the SOD1 ALS program does not impact Voyager’s other gene therapy programs; the Company continues to expect IND filings in 2025 from Neurocrine Biosciences for the program in ...
SOD1-ALS is a rare, progressive and fatal neurodegenerative disease that results in the loss of motor neurons in the brain and spinal cord. As ALS progresses, ...