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Push to get $3.5m drug approved to save kids' lives

Health
by Jane Hansen
Premium Content
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. ....

United States , New South Wales , Reena Mcintosh , Zarlee Brownlaw , Michelle Farrar , Greg Hunt , Montanna Brownlaw , Jonathan Casella , Aviana Mcelvee , Julie Cini , Mackenzie Casella , Nigel Hallett Reena Mcintosh , Katie Mcintosh , Ian Alexander , Kate Gough , Oakley Gough , Sam Ruttyn , Treatment Overseas Program , Therapeutic Good Administration , Pharmaceutical Benefits Advisory Board , Pharmaceutical Benefits , Associate Professor Michelle Farrar , Professor Ian Alexander , Prof Michelle Farrar , Christmas Day , Minister Greg Hunt ,

Push to get $3.5m drug approved to save kids' lives

Push to get $3.5m drug approved to save kids' lives
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United States , New South Wales , Reena Mcintosh , Zarlee Brownlaw , Michelle Farrar , Greg Hunt , Montanna Brownlaw , Jonathan Casella , Aviana Mcelvee , Julie Cini , Mackenzie Casella , Nigel Hallett Reena Mcintosh , Katie Mcintosh , Ian Alexander , Kate Gough , Oakley Gough , Sam Ruttyn , Treatment Overseas Program , Therapeutic Good Administration , Pharmaceutical Benefits Advisory Board , Pharmaceutical Benefits , Associate Professor Michelle Farrar , Professor Ian Alexander , Prof Michelle Farrar , Christmas Day , Minister Greg Hunt ,