“We were surprised at the rapid improvement of gross motor skills,” said Professor Eric Hoffman, one of the researchers who helped develop the drug. Duchenne muscular dystrophy is caused by mutations that prevent the body from properly producing dystrophin, a protein essential for maintaining healthy muscle. “The destructive processes in muscle start from birth,” Hoffman explained, even though symptoms typically aren’t recognized until the early school years. They were given either 2 or 6 milligrams of Vamorolone per kilogram of body weight each day for 12 weeks. After just 12 weeks of treatment, their average score had risen to around 8.