Canberra is subsidising a life-changing medication for children with dwarfism following its successful worldwide trial.
The drug, Voxzogo, allowed the “average bone growth resume”, targeting the underlying cause of the genetic condition Achondroplasia, dwarfism’s most common form.
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Led by Murdoch Children’s Research Institute’s Professor Ravi Savarirayan, the trial was so successful that it has received Australian government subsidy.
It would have cost families more than $330,000 a year for the medication, which is administered by a daily injection, however the subsidy has lowered that cost to just $30 a prescription, or $7.30 for concession card holders.
In an exclusive with the Herald Sun, Professor Savarirayan said the treatment has been given to children from birth and showed evident of reliving complications usually requiring surgery to fix.
“In this form of dwarfism, it’s like trying to drive your car with your handbrake on. It can’t reach average speeds and so the bones can’t grow normally,” Professor Savarirayan said.
“This medication releases that handbrake from a molecular point of view and allows average bone growth to resume.”
Voxzogo will not be a medication people will need forever – it will be administered until the skeleton stops growing and growth plates fuse, which is about when a person turns 18.
Around 150 children are born with dwarfism in Australia each year, and about 400,000 people around the world.
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