STEVE WILTON

Scientific Co-Founder

Professor Steve Wilton AO has a PhD from the University of Adelaide and extensive experience in molecular genetic studies.
Initially involved in finding disease-associated genes and then developing tests to confirm a clinical diagnosis and/or assist in genetic counselling, Steve has pioneered the use of antisense oligonucleotides to modify gene expression to by-pass catastrophic mutations that would otherwise result in Duchenne muscular dystrophy, a severe form of muscle wasting. The US FDA has granted accelerated approval for three “exon-skipping” drugs developed in his laboratory, eteplirsen, golodirsen and casimersen, as treatments for Duchenne muscular dystrophy.
Steve’s research team has become a pipeline of future drug development to treat many inherited and acquired diseases and is currently investigating treatments for more than 50 diseases, several of which are undertaken with national/international collaborators.

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