Charcot disease affects approximately 30,000 people in the United States. It causes progressive paralysis of the muscles, and generally causes death in less than five years.
New treatment is a “molecular stabilizer” that acts like a ‘stitch’ and forces the protein to stay in its correct configuration. The treatment successfully stabilized 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.