Can a drug cure a genetic disease? A thought-provoking piece on new treatments for genetic disease.
Steve is Director of the Perron Institute for Neurological and Translational Science and holds the Foundation Chair in Molecular Therapies at Murdoch University.
His research career has focussed on Duchenne muscular dystrophy (DMD), the most common and serious form of childhood muscle wasting, which arises from mutations in the dystrophin gene. His insights into the cause of DMD have led to an innovative treatment where the first of a series of drugs has just been granted accelerated approval by the FDA. This mutation specific approach promises to revolutionise treatments for DMD and many other genetic diseases.
Over the past twenty-five years, Steve and his team have made significant discoveries in their gene research and his work has been recognised with the 2012 Western Australian Innovator of the Year award; the 2013 Australian Museum Eureka Award for Translational Medicine; the 2014 LabGear Australia Discovery award; and most recently, he was a finalist in the 2016 Western Australian of the Year (professions category).