SMA

Close-up of newborn baby's feet poking out of blanket

Gene therapy for spinal muscular atrophy might have a high up-front price tag. But by screening and treating infants early, the therapy can save both lives and money in the long term.

New born baby

A trial has revealed that babies with SMA treated with gene therapy are meeting the normal developmental milestones of any baby.

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An international study involving UNSW and Sydney Children’s Hospital researchers has led to the first approved treatment for spinal muscular atrophy, a devastating genetic disorder in babies.