Billy Ellsworth completed his high school diploma in 2019 and is in college. This is extra special because Billy can still walk, due to a clinical drug trial in the USA, based on novel treatments created here in Western Australia.
This ground-breaking research led by Professors Steve Wilton AO and Sue Fletcher AO, has enabled kids like Billy with Duchenne muscular dystrophy to have hope for the future. Our dedicated researchers are committed to changing young lives, like that of Billy Ellsworth.
Billy was born with DMD; a fatal childhood condition that typically leads sufferers, usually boys, confined to a wheelchair before 12 years. Billy and his mother Terri travelled from Pittsburgh (USA) to Australia for the first time to spend time with the researchers bringing new hope to Duchenne suffers around the world.
“At the age of 11, Billy was unable to walk down a small bush incline in his hometown in Pittsburg without someone either side to stabilise him. Two years after starting the treatment, not only was Billy able to walk independently down the same path at a time when he should be in a wheelchair, his breathing has been stabilised and he can whistle as he walks up hill,” said Professor Wilton.
“In fact, his mobility and breathing improvement enabled Billy to complete the 2015 Pittsburg Children’s Marathon.”
Duchenne muscular dystrophy, occurring mainly in boys, is the most common childhood form of muscle wasting and is caused by a genetic error that prevents the body from producing dystrophin, a protein essential for maintaining muscle fibre strength and stability.
In the absence of dystrophin, muscle is much more susceptible to damage during regular activity, leading to loss of muscle and ultimately the ability to walk and breathe. Most children with Duchenne require a wheelchair before their early teens.
Billy, who is over 21 years of age and still able to walk, is receiving treatment with the first Duchenne drug, eteplirsen (Exondys 51) which received accelerated approval by the United States Food and Drug Administration (FDA) in 2016, developed by Professors Wilton and Fletcher and their team.
The second drug for Duchenne, golodirsen ( Vyondys 53), developed by the research team was approved by the US FDA in 2019, and in 2021, a third drug, casimersen (Amondys 45), was approved by the US FDA.