ALS News
7/23/20 - An experimental gene-silencing drug designed for a rare, inherited form of amyotrophic lateral sclerosis (ALS) has shown promise in a clinical trial conducted at the Emory ALS Center and other medical centers around the world.
The trial indicated that the “antisense” drug, known as tofersen, lowers levels of a disease-causing protein in people with a type of ALS caused by mutations in the gene SOD1. The results of the study, published in New England Journal of Medicine, have led to the launch of a phase 3 clinical trial to further evaluate the safety and efficacy of tofersen.
Although the study was primarily meant to evaluate safety and whether the drug lowers levels of the target protein, there are preliminary signs that high doses of tofersen slowed clinical progression of ALS. Read article here...
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