spinal muscular atrophy

newborn baby in hospital bed lying on their back with hospital bracelets on feet

Testing in the newborn period is more effective than conventional clinical diagnosis pathways for improving health outcomes in infants with the rare genetic disease.

Patient holding hands with doctor

Spinal muscular atrophy and cystic fibrosis are among the rare diseases being explored by UNSW Medicine & Health researchers.

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.