The Children’s Hospital at Westmead in New South Wales, Australia, is treating three boys in a clinical trial using a viral vector-based gene replacement therapy for Duchenne muscular dystrophy (DMD).
The trial aims to recruit ten boys aged younger than four years worldwide, who will receive a single-dose infusion.
These patients will be tracked for a minimum of five years for measuring the effectiveness of the therapy.
The viral vector-based therapy is designed to replace the faulty or mutated gene while targeting DMD at its root cause.
The Children’s Hospital at Westmead clinical trials medical lead Dr Michelle Lorentzos said: “This is the only trial in the world treating boys under four years of age ‒ we think by treating the boys earlier, we may be able to prevent much of the weakness and disability that has already occurred in older patients.
“If successful, this treatment could change the landscape of treatment for boys with DMD by offering a transformative intervention that may enable the boys to continue walking into adulthood and also improve their life expectancy.”
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By GlobalDataThe trial was enabled by the Kids Advanced Therapeutics Program at SCHN, which is supported by Luminesce Alliance and the Sydney Children’s Hospitals Foundation.
The Children’s Hospital at Westmead Clinical Genetics staff specialist and trial principal investigator Kristi Jones said: “The fact we have been able to run this trial and are the first site in the world to do so, is a remarkable achievement and the result of many years of hard work.
“This wasn’t something we would have been able to achieve on our own and we give our sincere thanks to our supporters, like Save Our Son Duchenne Foundation, who have supported our research and teams from the beginning ‒ their advocacy has been instrumental in making this trial possible.”
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