Green light for Australian arm of Sanfilippo gene therapy trial

Amazing news announced overnight of an Australian clinical trial for children suffering the fatal genetic Sanfilippo has been approved!  

This is a true story of people powered medicine. When Meg Donnell’s children were diagnosed with this catastrophic fatal condition a few years back, she made it her life’s work to fund and fight for a cure.  Now they have hope!

The Sanfilippo Children’s Foundation is thrilled to today announce that 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 South Australia.

Megan Donnell, Founder and Executive Director of the Sanfilippo Children’s Foundation, said  TGA approval is a significant milestone. “Following from this exciting progress we are now looking forward to news that the trial is commencing.”

Ms Donnell said that for Australian families with children battling this devastating neurodegenerative disease, clinical trial participation offers a rare opportunity to access an experimental therapy, giving children with this aggressive condition the hope of an effective treatment.

“Children whose parents were once told that treatment was at least a generation away now have a small glimmer of hope. This means some Australian children [who meet the eligibility criteria] will receive an experimental treatment and potentially face a different future than the one they were handed upon diagnosis.”

For more information or to make a donation visit: www.sanfilippo.org.au.


Author: Laurice Klaire

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