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World-First trial offers hope to children with Duchenne Muscular Dystrophy

MUSCULAR DYSTROPHY words written on label tag with medicine,syringe,keyboard and stethoscope with wood background

Three boys in NSW have become the youngest patients in the world to receive therapy for Duchenne Muscular Dystrophy as part of a world-first international clinical trial for children aged under four. 

Duchenne muscular dystrophy (DMD) is a rare and life-limiting genetic condition mostly affecting boys that causes rapid muscle weakness and results in almost all patients needing a wheelchair by 12 years of age.

There is no known cure.

The trial will recruit 10 boys under four years of age worldwide, with patients followed over a period of at least five years to measure the therapy’s effectiveness, including three in NSW who are being treated at The Children’s Hospital at Westmead.

The DMD clinical trial will use a novel viral vector-based gene replacement therapy to target DMD at its root cause, replacing the faulty or mutated gene with a healthy version in a single-dose infusion.

Current management of DMD involves high-dosed steroids, combined with physical therapy and allied health support but while it can lead to some improvement, it is also associated with difficult side effects and is not a long-term treatment.

Gene replacement therapy has already shown success in treating other genetic conditions, including spinal muscular atrophy (SMA), a condition causing rapidly progressive muscle weakness and early death in children.

NSW Premier Chris Minns said: “This new gene therapy represents a beacon of hope for these three young boys and their families.”

“I’m very proud that NSW is a part of the world trial for this debilitating disease.”

“The Sydney Children’s Hospitals Network has the expertise and capability to deliver world-leading paediatric clinical trials like this, which can lead to major clinical improvements for these children.”


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