Health Australian drug helps US Duchenne sufferers

Australian drug helps US Duchenne sufferers

Sixteen-year-old Billy Ellsworth suffers from Duchenne – a fatal condition that leads to severe muscle weakness and which typically puts kids, usually boys, in a wheelchair by the time they’re 12.

But the US teenager is still walking thanks to a new drug developed by researchers from Murdoch University.

The drug, Exondys 51, is the first of its type approved by the US Food and Drug Administration (FDA) and addresses the underlying genetic causes behind Duchenne.

Exondys 51 was developed by professors Steve Wilton and Sue Fletcher and their team at the Perron Institute.

“At the age of 11, Billy was unable to walk down a small bush incline in his hometown in Pittsburgh without someone either side to stabilise him,” said Professor Wilton.

“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.

“In fact, his mobility and breathing improvement enabled Billy to complete the 2015 Pittsburgh Children’s Marathon.”

Professor Fletcher said Exondys 51 is not a cure but reduces the severity of the Duchenne.

“Loss of the ability to walk is a significant milestone in the progression of the disease, so keeping boys on their feet is crucial,” Professor Fletcher said.

“If we can keep boys moving, keep them upright, this reduces contractures, improves breathing and maintains independence and the health and wellbeing of these kids.”

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