4 April 2021 - Parents whose children have died or suffer from spinal muscular atrophy are fighting to make breakthrough gene therapy — which costs $3.5 million for a one-off dose — affordable, and to have all Aussie kids screened.
It is one of the most expensive drugs to ever hit the market, but at $3.5 million per one-off dose, it can mean the difference between life and death for a baby born with spinal muscular atrophy.
Zolgensma, a gene therapy, is approved by the Therapeutic Good Administration but is currently facing a Pharmaceutical Benefits Advisory Committee decision on whether to list it for subsidy on the Pharmaceutical Benefits Scheme.