Parents secure miracle $3 million treatment for sick toddler
AFTER a year of "ups and downs", a family's wish for their daughter Wynter has come true - she has finally been administered the $3.1 million treatment likely to change her life.
Diagnosed with Spinal Muscular Atrophy type 1 at four-months-old, Wynter was in need of Zolgensma, an expensive gene therapy medication not yet publicly available in Australia.
Her parents, Meringandan couple Kellee and Jamie Clarkson, faced many hurdles trying to source the treatment, but were buoyed by support of the community.
"It was a rollercoaster of emotions," Ms Clarkson said.
"However some people went out of their way to help us and without them we would have felt alone in this journey."
Although warned of unfavourable odds, the family entered an application to the Department of Health's Medical Treatment Overseas Program in March.
With backing and support from One Nation, MTOP approved the application within a week - but another obstacle awaited.
"With coronavirus, we were knocked down again, because we needed to travel to America for treatment," Ms Clarkson said.
"However, we applied for a special act of grace, where the drug would be imported to the country if the government agreed - and they did.
"We were truly grateful (for the decision)."
The government's approval was made all the more incredible by its timing, as Wynter was four-months off being too old for the treatment.
At the end of May, the family drove down to a hospital in Sydney, where Wynter received the Zolgensma treatment.
"It's a huge thing to dream about something and then have it happen - we couldn't believe it," Ms Clarkson said.
"Not only has it taken away a lot of pain for Wynter, it has also given us a new beginning as a family - we know that we have done the best we could as parents."
Ms Clarkson said although long-term outcomes were still unknown, most children responded well in clinical trials.
"The distress and trauma Wynter has experienced in the first two years of life has been excruciating - we're hoping that this a one-off treatment for life," she said.
"And this isn't just for Wynter - it's for other (SMA) families with children under two.
"I want to thank everyone on the Wish for Wynter Facebook page for believing in us."