The moving story of Manly siblings Isla and Jude, who are living with the fatal genetic Sanfilippo Syndrome, has touched the hearts of families across the country. But today, there is cause for hope for this beautiful family.
Isla is seven years old. She loves dress-ups, singing and playing with her brother. Isla is looking forward to starting school next year. Jude is her younger brother, five years old. He is full of affection and boundless energy. Jude makes friends wherever he goes.
But while they sound like your typical, healthy siblings, both Isla and Jude have the fatal Sanfilippo Syndrome. Soon, their health will begin to deteriorate at a rapid pace. Their bodies will lose the ability to break down cellular waste causing severe brain damage and ultimately a painful and premature death. Without treatment they will not live.
But finally, there is hope for this fighting family. Overnight, an Australian clinical trial for children suffering the fatal genetic Sanfilippo has been approved.
The US-based biopharmaceutical company, Abeona Therapeutics, has received regulatory approval to initiate a clinical trial in Australia with ABO-102 gene therapy for patients with Sanfilippo Syndrome type A (aka MPS IIIA). The clinical study was approved by the Australian Government’s Therapeutic Goods Administration (TGA) and the company is conducting the phase 1/2 clinical study at the Women’s and Children’s Hospital, Adelaide, in SA.
This is a true story of people powered medicine. When the children were diagnosed with this catastrophic fatal condition a few years back, their mum Meg made it her life’s work to fund and fight for a cure. Now they have hope. But while this is a step forward, the fight isn’t over.
We are pleased to support this incredible cause because we understand the amazing force of people power, and when we are share this story and donate, we can work together to hopefully save these little lives.