Billy Ellsworth now in college in the United States

Exondys 51 treatment slows the progression of Duchenne Muscular Dystrophy

18-year-old Billy Ellsworth, a participant in the Exondys 51 (eteplirsen) Duchenne muscular dystrophy clinical trial in the United States, is still walking and now in college.

Three years following US FDA approval of the first drug Exondys 51, Billy maintains stability and defies his disease by not being confined to a wheelchair. Billy’s mum Terri Ellsworth is grateful to Professors Steve Wilton and Sue Fletcher who pioneered the treatment at the Perron Institute and The University of Western Australia. The Exondys 51 treatment slows the progression of Duchenne Muscular Dystrophy. It has allowed her son to continue walking well beyond expectations. Children with Duchenne can start losing their ability to walk around the age of 8.

“When Billy was diagnosed almost 15 years ago, I really didn’t expect that I’d witness him walking to receive his high school diploma. But he graduated and walked well and confidently with his peers to receive his diploma and with honor cords. He is now taking college classes and is about to finish his first semester of college,” Terri Ellsworth said.

Over two years ago, for the opening of the Sarich Neuroscience Research Institute building where the Perron Institute is housed, Billy and Terri were able to visit Australia for the first time. They and Professors Wilton and Fletcher were interviewed in a short video filmed on the Swan River. Watch it here.

Terri described her hopes for Billy to stay the course, and if he did, she fully expected to see him walk across the stage in two years to receive his high school diploma.

Reflecting on achieving her hopes for the future, Terri said: “Well that came and went and he is still going strong! I think this is quite remarkable and noteworthy! I am so grateful to the Perron Institute, UWA and Murdoch University.

“Billy is now in college classes and walks into that campus facility as well. I think it is important, motivational and inspirational to see Billy walking well at almost 19 years of age.

“It’s critical to keep the science moving forward as quickly as possible to help as many patients as possible with other mutations, and most importantly to help rescue cardiac muscle for meaningful quality of life and longevity.”

The second drug, Vyondys 53 (golodirsen) received US FDA accelerated approval in December 2019.

Pictured: Billy Ellsworth graduates 2019