A Sunshine Coast newborn has become the first Queensland child to undergo gene therapy for spinal muscular atrophy (SMA) in their home state.
Queensland Children’s Hospital is now accredited to provide the promising new therapy, meaning patients and families no longer need to travel interstate for the treatment and can continue to be supported by the specialist care team they have known since diagnosis.
Two-month-old Layla Christensen was diagnosed with SMA in February 2023 after her parents noticed she was floppy, never kicked her legs well and had reduced movement compared to her older brother.
SMA is a genetic disorder that damages the motor neurones in the spinal cord which control the body’s ability to move, breath, cough and swallow.
About 1 in 10,000 children born in Australia are affected by the condition.
Untreated SMA is the number one genetic cause of death for children under the age of two.
On 23 March 2023 Layla became the first patient to have onasemnogene abeparvovec (Zolgensma®) administered at the Queensland Children’s Hospital.
Zolgensma® is a gene therapy that targets the genetic root cause of SMA by replacing the function of the missing or nonworking SMN1 gene.
The therapy is given as a single dose intravenously over 60 minutes and can stop the progression of SMA. The treatment is not a cure for SMA and cannot reverse damage already caused by the condition.
Zolgensma® is one of three treatments available for SMA in Queensland. Nusinersen (Spinraza®) and risdiplam (Evrysdi®) are also effective treatments but need to be taken for life.
Queensland Children’s Hospital consultant paediatric neurologist Dr Anita Cairns said Layla was doing well after the therapy.
“We have already seen improvement in Layla’s movement since commencing Spinraza® at the time of diagnosis. We would anticipate seeing ongoing gains from Zolgensma® in the next few weeks with improvement in head control and gains in motor skills,” Dr Cairns said.
Layla’s father Troy said the treatment would change Layla’s life and meant the family would not have to visit the hospital every month.
“Layla can now worry about regular kid problems and can live her life to the fullest. We can enjoy things like a family holiday without worrying about how close we are to the hospital,” he said.
The Queensland Children’s Hospital became an accredited treatment centre for gene therapies such as Zolgensma® in early March 2023. Nine other Queensland Children’s Hospital patients have received this treatment but were treated in Sydney.