An Australia-first clinical trial to try to extend and improve the lives of children with the paediatric equivalent of motor neurone disease (also know as spinal muscular atrophy) has treated its first patient in Victoria.

An international clinical trial of a new gene therapy, called antisense oligionucleotide, is taking place at the RCH in the hope of prolonging and improving the quality of life of affected children.

Seven-month-old Emilia Hlusko yesterday became the first Victorian patient – one of only 120 globally – to be given the spinal cord injection.

This is a ‘double-blind’ trial, so neither her doctors nor Emilia’s mother, Liz Gleeson, know if she has received the gene therapy or a placebo.

Liz said her aim was to help other parents avoid her heartache, and hopefully to allow Emilia to enjoy more time with her brother Tommy, aged two years.

Photo of Emilia and Liz courtesy of the Herald Sun.