Early vamorolone treatment shows promise for toddlers with Duchenne muscular dystrophy

Treating boys with Duchenne muscular dystrophy before symptoms start may lead to major benefits, a new Binghamton University study finds.

Researchers tested the drug Vamorolone, FDA-approved in 2023, in 20 boys ages 2 to 4 who had never taken steroids. After 12 weeks, gross motor skills improved rapidly — especially at the higher dose. On a motor scale where healthy kids score around 10, boys with DMD went from an average of 5 to 8.“There were no serious side effects,” said researcher Prof. Eric Hoffman. Growth was not impaired, unlike with traditional steroids.

DMD damage begins at birth, but diagnosis often comes years later. With DMD now added to U.S. newborn screening, doctors may soon be able to start treatment much earlier.“Hopefully, vamorolone may become an option for these babies if these preliminary data are confirmed,” Hoffman said.

The study was small and needs larger trials, but suggests early intervention could help protect muscle before it’s lost.