Professor Steve Wilton is the Perron Institute’s Director and the Director of the Centre for Molecular Medicine and Innovative Therapeutics at Murdoch University. With Professor Sue Fletcher, he also leads the Molecular Therapies Laboratory at Murdoch University.
Professor Wilton joined the institute in 1991 and with his colleague Professor Sue Fletcher pioneered the development of a radically new therapy for the devastating childhood disorder, Duchenne muscular dystrophy. In September 2016, the USA Food and Drug Administration (FDA) gave accelerated approval to a new treatment for Duchenne created by Professor Steve Wilton, Professor Sue Fletcher and their team. Eteplirsen (Exondys 51) is the first dystrophin restoring drug of its type ever approved by the FDA. In December 2019, the FDA granted accelerated approval for golodirsen (Vyondys 53), targeting another subset of Duchenne. In February 2021, the third FDA accelerated approval was granted for casimersen (Amondys 45). The three drugs developed by Professors Wilton and Fletcher and their team, can treat almost thirty per cent of patients diagnosed with Duchenne.
Current research explores the use of similar drug strategies in other diseases, including spinal muscular atrophy, cystic fibrosis and Pompe’s disease to name a few.
Professor Wilton and Fletcher’s research on Duchenne has been acknowledged through several awards, including the 2012 WA Innovator of the Year Award, the 2013 Eureka Prize for Medical Research Translation and the 2014 Labgear Australia Discovery Science Award. In 2016 Professor Wilton was nominated Western Australian of the Year in the Professions category.