Gene therapy access expands for Australian children with muscle disease
Zolgensma, a $2.5 million gene therapy, is now available to more Australian children with spinal muscular atrophy.
Why it matters
This expands access to a treatment that can be genuinely life-changing for kids facing a terminal disease.
Key facts
- $2.5 million
- Cost per dose
- Pharmaceutical benefits scheme
- Most expensive drug
- Around 1 in 10,000 babies
- Incidence of SMA
- Treated at 4 weeks, now 8 years old
- First treated case
- Eligibility has expanded from babies up to 9 months to children weighing up to 21 kilograms.
- Spinal muscular atrophy damages nerves that control muscles, leaving children unable to move, swallow or breathe.
- The therapy delivers a working copy of the faulty gene.
- One girl was treated at four weeks old and is now eight with all developmental milestones reached.
- Health Minister Mark Butler confirmed it is the most expensive drug on the pharmaceutical benefits scheme.
