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Faster access to new treatment adds centimetres to height

A new treatment which adds many centimetres to the height of children living with achondroplasia is now readily available at Sydney Adventist Hospital – without the long waiting lists. This is potentially life changing because the earlier the treatment starts, the more height can be gained.

Achondroplasia is a genetic condition that impedes growth, predominantly inhibiting growth of the long bones in the body such as the legs and arms. “It affects one in 20,000 live births, and children with achondroplasia grow to an average adult height of 120cm,” said Dr Neil Ginsberg, the first paediatrician to administer Vosoritide at the San.

The new drug, Vosoritide, was only released on the Pharmaceutical Benefits Scheme (PBS) in Australia last year. “Vosoritide blocks the process that is inhibiting the growth of long bones in children with achondroplasia. The studies conducted on this drug globally have been quite exceptional, showing that children can achieve up to 2cm of extra growth in height per year,” said Dr Ginsberg. “That means if treatment is started early and continues, for example, for 16 years, this can add an extra 30cm to their average growth height – from 120cm to potentially 150cm.” 

A momentous day

When 14-month-old toddler Finn* had his first injection of Vosoritide at the San, it was a big day for him, his parents, and his doctor. “We found out through scans at 30 weeks gestation that Finn had achondroplasia, and it was obviously a bit of a shock for us,” said parent Todd. “We did a lot of research and we knew a new drug was coming but it hadn’t been approved on the PBS in Australia at that time. Once it was approved, we found out there were long waiting lists in some places to start treatment. That was stressful, because waiting another year to start treatment makes a big difference to a child who is already small, because each year can mean a few centimetres in height.”

“Then we heard about Dr Ginsberg offering this at the San. It’s absolutely fantastic; life changing. I get shivers down my spine when I think of this being available for Finn. I’m sure he will appreciate it one day too, even though he doesn’t like the injections just now,” added Todd.

Earlier the better

While it is great news that Vosoritide is now available on the PBS, there are currently long waiting lists in some States. “The protocol is that the first dose of Vosoritide has to be given in hospital, and within the public system there is quite a backlog of patients,” noted Dr Ginsberg. “There is concern they’re only going to be able to get through all of them in about 18months to two years. Commencing Vosoritide early is key. The earlier the treatment is started, the more growth and better results you’re going to get.”

“The San was the first site in Sydney to offer Vosoritide outside the Children’s Hospitals that is offering Vosoritide,” added Dr Ginsberg. “This new treatment is very exciting; it gives families an outcome they never previously thought possible for their child when they were initially diagnosed. Our nursing staff on the paediatrics ward at the San have been trained to administer the first dose, observe them, and likewise train the child or parents for the ongoing administration. We are very excited to offer this service and to help families access this novel treatment without the long waiting time.” 

For Dr Ginsberg, being able to provide this new treatment for his young patients is a special time in his career. “I think it is incredible. Throughout your medical career you have a few life-changing interventions that come onboard that didn’t exist before. I think it’s phenomenal to now be able to sit down with parents – when a newly diagnosed child comes along – and say ‘Look, last year I couldn’t have offered this treatment, but this year things are very different’. Having the option of Vosoritide now is an amazing intervention.”

“There will always be some who will be uncertain of new interventions and that is quite acceptable. Every family needs to have the information and make the decision they are comfortable with. For those who are keen to pursue Vosoritide, it offers them a lot of hope. Here at the San it has been great to be involved in something that is giving such a positive outlook for families living with achondroplasia,” added Dr Ginsberg.