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Novel therapy trial for kids with Duchenne Muscular Dystrophy

Three boys from New South Wales, Australia have been recruited to participate the world first trial for kids aged under four with Duchenne Muscular Dystrophy.

Duchenne muscular dystrophy (DMD) is a rare and life-limiting genetic condition primarily observed in boys. It is characterized by progressive muscle weakness, which typically begins in early childhood. There is no cure at the moment.

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.

The DMD clinical trial was enabled by the Kids Advanced Therapeutics Program at SCHN, a program kindly supported by Luminesce Alliance and Sydney Children’s Hospitals Foundation which aims to deliver clinical trials of advanced therapeutics and to speed up translation into clinical care.


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