A new drug for the treatment of a rare and fatal muscle wasting disease, developed by West Australian researchers, has been approved by the US Food and Drug Administration.
University of WA deputy vice-chancellor of research Robyn Owens said the drug, Eteplirsen, offered new hope for sufferers of Duchenne muscular dystrophy, who without treatment were usually confined to a wheelchair before the age of 12 and had a life expectancy of 30.
"It's the first treatment for DMD that addresses the cause of the disease and the hope is that it will slow its progression and keep patients mobile for longer," Professor Owens said.
Duchenne muscular dystrophy affects one in 3500 boys worldwide, causing deterioration of every muscle in a sufferer's body, including the heart, and eventually leads to death.
It is very rarely suffered by females, affecting about one in 50 million girls.
Murdoch University professors Steve Wilton and Sue Fletcher developed the treatment at UWA's Centre for Neuromuscular and Neurological Disorders, the WA Neuroscience Research Institute and Murdoch University's Centre for Comparative Genomics.