Push to get $3 5m drug approved to save kids lives
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Push to get $3 5m drug approved to save kids lives | Fraser Coast Chronicle
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Push to get $3 5m drug approved to save kids lives
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Health by Jane Hansen
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Subscriber only 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 to paralysis, breathing difficulties and death within months for babies born with Type I, the most serious form. About 30 babies a year, an entire classroom of children, are born with SMA each year.