Associate professor Andrew Kornberg is flying for funds. Photo: Fly for the Kids.
A dedicated Australian doctor and pilot with a bold goal has landed in Esperance as part of his fundraising mission to support children with rare and complex diseases.
Senior Neurologist at the Royal Children’s Hospital (RCH), Associate Professor Andrew Kornberg, is flying his plane with the aim of raising $4.5 million for gene therapy-related infrastructure and resources.
“It is for rare diseases, which is a disease less than one in 2,000 but if you put all the diseases together it’s about 10 per cent of the population,” Prof. Kornberg said.
“If a child has a rare disease, a third of them won’t make their fifth birthday.”
It is the second time Prof. Kornberg has merged his passions to help others, after a similar fundraiser in 2017.
He said the community could “co-pilot” his three-week journey by spreading the word, educating themselves on the diseases and donating.
“Everyone is born and everyone dies but I think what you do in between is what’s important and this is what’s in between for me, it really is important to get this done,” he said.
“What inspired it was two children I look after with muscular dystrophy — there’s no gene therapy or cure at this stage but we were offered to be part of an international trial gene therapy.
“Unfortunately, at that time we didn’t have the infrastructure in place, it was the middle of winter, beds get tied up for weeks so we couldn’t do the gene therapy.
Everyone is born and everyone dies but I think what you do in between is what’s important and this is what’s in between for me, it really is important to get this done.
Associate Prof. Andrew Kornberg.
“I realised then we needed a dedicated place with beds and infrastructure to allow children with rare conditions to be provided with leading edge treatments.”
He said gene therapy had come a long way but was still expensive and often could not be delivered in a sustainable way.
He said treatment could change a child’s life.
“A little boy called Axel has a condition called spinal muscular atrophy and eight years ago there was no treatments,” he said.
“Now we do newborn screening so we can treat them before they have symptoms and Axel is a normal boy —walking, running around just like any other kid of his age.”
Prof. Kornberg said he was flying from Ceduna in SA to Esperance.
He said Esperance was more of a pit stop on his agenda to “eat, refuel and get ready for the next day.”
Prof. Kornberg said in some locations he would be visiting patients who had benefited from the treatment.
