Health
Push to get $3.5m drug approved to save kids’ lives – Fraser Coast Chronicle
Parents are fighting to make breakthrough gene therapy — which costs $3.5m 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 (SMA).
Zolgensma, a gene therapy, is approved by the Therapeutic Good Administration (TGA) but is currently facing a Pharmaceutical Benefits Advisory Board (PBAC) decision on whether to list it for subsidy on the Pharmaceutical Benefits Scheme.
One in 35 people carry the gene mutation for SMA.
SMA leads…
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