Leading the world in gene therapy research for spinal muscular atrophy
A world-leading research program has seen the Sydney Children鈥檚 Hospital, Randwick, be the first hospital globally to deliver a cutting-edge gene therapy for children up to six years old with spinal muscular atrophy (SMA).
SMA is a rare genetic disease causing progressive muscle weakness. Without intervention, the most severe types of SMA can have a life expectancy of less than 2 years old.
The international clinical trial, dubbed the SMART trial, investigated...
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