Six-year-old Beau Cosgrove, of Taralga is one of 20 children across Australia who will receive free life-saving treatment for their rare medical condition.
Beau is living with Morquio A Syndrome. It’s a condition he has had since birth, and the only available treatment, Vimizim (elosulfase alfa), costs about $400,000 a year.
On Friday Federal Hume MP Angus Taylor confirmed the government would make Vimizim available under the Life Saving Drugs Program from August 1, this year.
“It’s an awful disease that cruelly affects children like Beau,” Mr Taylor said. “I know having access to Vimizim will be life-changing for Beau and his parents Joe and Sam, his younger twin brothers Zac and Ty, and younger sister Kathryn.”
Morquio A Syndrome, or mucopolysaccharidosis (MPS) type IV A is an inherited metabolic condition.
People born with it are either missing, or do not have enough of, a crucial enzyme needed to break down long chains of sugar molecules. As a result they have abnormal development and a possible early death.
Vimizim is the first and only available enzyme replacement therapy (ERT) for patients with Morquio A.
Beau’s mum Sam described the diagnosis of Morquio A as like “mourning” the loss of your child – it’s a condition which stops children being children, “no sports, no nothing”.
Sam knew that a clinical trial was the only option for Beau and registered him for access to ERT in late 2012. A year later Beau was accepted onto Dr Kaustuv Bhattacharya’s drug trial at The Children’s Hospital at Westmead in Sydney.
“ERT has been a miracle for Beau. The difference in him, on the treatment, has been remarkable,” she said. “He’s been able to go to a mainstream school and will be able to contribute to society.”
While Beau will never be cured, ERT provides people living with Morquio hope for the future.