A disabling genetic disease, Duchenne muscular dystrophy is the most common inherited neuromuscular disorder among children.
The ABC interview follows recent developments in the USA where advisers to the Food and Drug Administration (FDA) narrowly recommended that the agency approve the first gene therapy for the disease. A final decision is expected to be made by the FDA by the end of May, 2023.
Professor Rasko says that over the past five years, there have been over a hundred cell/gene therapies approved globally for diseases that include haemophilia, thalassemia, spinal muscular atrophy and inherited blindness. Nine cell/gene therapies have been approved by Australia’s TGA since 2018.
The full interview can be heard online on the Radio National website.
Information about Professor Rasko and his research can be found on the Centenary Institute website.
Further reading: Approved gene therapies in Australia: coming to a store near you.