‘I think Jesse would be pretty proud’: Major milestone in the fight against Duchenne muscular dystrophy

A new drug has been approved that will help improve the quality of life for young patients with Duchenne Muscular Dystrophy. CTV London’s Reta Ismail reports.

A major milestone in the fight against Duchenne muscular dystrophy is bringing new hope to families across Canada.

Health Canada has approved a new drug called Agamree, also known by its scientific name, vamorolone, making it the first and only therapy authorized in Canada to treat Duchenne muscular dystrophy (DMD) in children aged four and older.

The announcement marks a deeply personal victory for John Davidson, whose son Jesse was diagnosed with the rare genetic disorder in the early 1990s.

Davidson has since spent decades advocating for research and treatment through Jesse’s Journey, now known as Defeat Duchenne Canada.

“This is what we wanted all along,” Davidson said. “When we set out to try to make a difference in the lives of all those boys with Duchenne, I think Jesse would be pretty proud of what’s happened today.”

Duchenne muscular dystrophy affects approximately one in every 5,000 boys. The disease causes progressive muscle degeneration and has no known cure, and treatment options have been extremely limited.

Agamree is a steroid-like medication specifically developed to treat DMD. According to Dr. Craig Campbell, a pediatric neuromuscular specialist at the London Health Sciences Centre, it offers similar motor benefits to traditional corticosteroids but with fewer side effects.

“This is a drug that was developed with Duchenne muscular dystrophy in mind,” said Campbell. “The hope, and what we’ve seen in the evidence, is that we can get the same effect on motor improvements but reduce some of the really problematic side effects.”

Defeat Duchenne Canada played a pivotal role in Agamree’s development, providing a $1-million research grant that helped launch the clinical trials. Though the exact cost to Canadian patients is still unknown, Campbell said the approval marks a turning point.

“There’s a lot of hope on the horizon with genetic-based therapies and even gene therapies that are coming to the forefront for Duchenne,” he said.

For Davidson, the news represents more than just a regulatory green light, it’s a validation of years of effort, hope, and perseverance.

“If he were here today, he would probably say, ‘Well, it’s about time.’ And then he would say, ‘This is great news,’” Davidson said of his son Jesse.