A vitamin could help treat Duchenne muscular dystrophy

Researchers are working on a new strategy to combat one of the most severe forms of muscular dystrophy. Rather than acting on the defective gene, they are using large doses of a vitamin. Duchenne is the most common and severe form of muscular dystrophy. Because of this genetic disease, one out of every 3,500 children spends their 12th birthday in a wheelchair. This disorder progressively leads to general paralysis, and most patients die of respiratory failure. The disease is caused by a genetic mutation that prevents a protein required to keep muscle cells intact from being produced. While most research focuses on repairing the defective gene, researchers at EPFL have come up with a different strategy.