New treatment shows promise against fatal neurological disease: study

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Only a mice study under very artificial conditions, so it is early to think it will be useful for sure, but at least it seems the concept is realistically possible, best case scenario this drug can be used effectively on humans, but even if not, it still means this could be done with another compound better designed for the human disease.

Agar said he hoped it might become a co-treatment for Biogen's Qalsody, a breakthrough regimen that received accelerated approval by the Food and Drug Administration in 2023, which works by reducing the number of SOD1 gene copies the body produces.

The last part is mistaken, the drug do not modify in any way the genes, it works by reducing the number of copies of the protein produced from the gene.

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