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A 13-year-old boy’s dream of keeping up with his friends on the soccer field could soon become a reality after he gained access to a life-changing dwarfism medication at a Sydney hospital.
Patients with a common form of dwarfism called achondroplasia are able to access the newly subsidised drug Vosoritide at The Children’s Hospital at Westmead to help them grow taller.
Riley Schroder is the first patient to access the medicine and hopes it will allow him to live his sporting dreams.
“I wanted to get taller, and play on the big soccer field and keep up with my friends,” Riley said.
His mum Lousie Martin was overwhelmed with the medical breakthrough.
“I just went to tears, happy tears,” Martin said.
She said it would help him to reach things, be able to drive, have more chance of employment and assist with other health issues.
“Riley’s hoping to get off his CPAP machine which he’s been on since birth,” Martin said.
Minna Koo, a doctor at The Children’s Hospital at Westmead, said the only previous option was to have limb-lengthening surgery, a long and painful process.
The disorder is caused by a mutation in the FGFR3 gene that slows bone growth.
Vosoritide works on a receptor to silence that activity and stimulate growth.
It can double growth velocity from three centimetres a year to six.
“Their expected adult height has been increased by 10 centimetres or more depending on when they start the treatment,” Koo said.
“It’s been an amazing outcome for these children.”
The treatment is administered daily, is well-tolerated and works directly on the growth plates of the bones.
It is only suitable for children who are still growing and aged from two years old to 18.
Koo said it was a personal choice for each child and family.
Time will tell whether it can prevent long-term complications such as breathing and hearing issues or the need for spinal surgery.
International trials led by doctors at Westmead and at the Murdoch Children’s Research Institute were pivotal in getting the drug listed on the PBS and enabling 300 children – like Riley – to benefit.
