It has been suggested that mitochondrial dysfunction is among the earliest cellular deficits of mdx muscles, and that mitochondria in dystrophic muscles don’t respond well to injury.
Raxone (Idebenone) is being developed by Santhera Pharmaceuticals, and is designed to slow the rate of respiratory decline in patients with Duchenne. Raxone is aimed at those patients who are over 10 years old and showing respiratory problems, and not taking steroids. It should be noted that Raxone is not designed as a replacement for steroids.
Raxone works to increase the energy output of the cell’s mitochondria. It achieves this through acting as an electron carrier, delivering more electrons to the mitochondria enabling the production of more energy. In addition to helping out the power output of the cell, Raxone acts as an antioxidant protecting the cell from oxidative stress and damage which occurs as a result of inflammatory responses. Through targeting mitochondrial dysfunction in Duchenne, Raxone helps to prevent cell death and muscle degeneration. This agent could become a good option for patients who are unable to tolerate the side effects of glucocorticoids.
