Posted by Michael Wonder on 05 Nov 2017
New hope for families affected by spinal muscular atrophy, with TGA approving drug
4 November 2017 - A drug that can extend the life of children and adults with spinal muscular atrophy (SMA) has been approved by the Therapeutic Goods Administration and is now commercially available.
Until now, sufferers of SMA had few options, and babies with the most severe form of the genetic disease, SMA1, deteriorated until they couldn't swallow and breathe – usually by nine months of age.
Julie Cini, chief executive of SMA Australia, said the TGA decision was "exciting" news and the next step was to see it listed on the Pharmaceutical Benefits Scheme.
"We've campaigned so hard for this as it shows fantastic promise, but a PBS listing would be the icing on the cake," said Ms Cini, who lost two baby daughters to the disease a decade ago.
