Three boys in NSW have become the youngest in the world to receive therapy for Duchenne muscular dystrophy as part of a world-first international clinical trial for children aged under four.
Lucas (3), Hunter, (4) and Ollie (3) were all diagnosed with Duchenne muscular dystrophy before their third birthdays. The rare and life-limiting genetic condition affects 1 in 5,000 boys and causes rapid muscle weakness, resulting in almost all children needing a wheelchair by 12 years of age.
Current management involves high-dosed steroids, combined with physical therapy and allied health support, but there is no long-term effective treatment and no known cure.
