The difference John deserves

Diagnosed before birth with a severe neuromuscular disorder known as spinal muscular atrophy (SMA), John entered the world under the heavy shadow of a condition that had previously taken the life of his sister, Hannah.

Carolyn and Steve welcomed their daughter Hannah in 2014. Just eight months and 22 days later, the two had to say goodbye.

At the time, there was no way to treat SMA, and the disease progressed relentlessly. For the Pearce family, the first clinical trials to treat SMA began shortly after Hannah’s passing – a bittersweet realisation that a potential treatment was just out of reach for their daughter.

In 2023, the two welcomed John into the world. But this time, unlike with Hannah, there was a way to stop the disease.

At just five days old, John was treated with a ground breaking gene therapy infusion known as Zolgensma.

John is now 19-months-old and thriving. Beyond a sleeping mask, the therapy has had a minimal impact on his daily life – allowing him to grow and develop just like any child his age.

In Australia, about 400,000 children live with rare or complex illnesses like spinal muscular atrophy. 

Until recently, families across Queensland often faced the tough choice between missing out on treatment or travelling interstate or overseas to access a trial.

With the support of the Children's Hospital Foundation, the Queensland Children’s Hospital ACTION Centre launched in May to provide specialised care - close to home.

This new statewide hub brings advanced treatments and clinical trials to children with life-limiting conditions, just like John.