Dystrophin protein (green) is deficient in DMD, and muscles fall apart.
A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. Boys 6 years and older can take Duvystat, to slow the course of the illness. FDA classifies it as a "nonsteroidal treatment" – not a gene therapy, but it affects gene expression.
To continue reading, go to DNA Science, where this post first appeared.