Charcot disease is fatal and for the moment without a known effective cure. The new treatment is a "molecular stabilizer" that acts like a "stitching" and forces the protein to stay in its correct configuration.
It succeeded in stabilizing 90% of SOD1 proteins in blood cells and 60 to 70% in brain cells. The researchers now hope to obtain approval to move on to clinical trials in humans.. advance marketing authorization was issued in April 2023 in the United States for a drug (Qalsody from the Biogen laboratory) targeting certain forms of Charcot disease.