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A family is trying to raise over $2 million to give their baby a gene therapy treatment to save her life - CNN
Jul 11, 2020 1 min, 48 secs
The family is trying to raise $3 million Canadian dollars.

"Gene therapies such as Zolgensma have the potential to reduce the financial burden on patients, families and the overall healthcare system by replacing repeat, lifetime therapies with a single treatment," a spokesperson for AveXis, the company that markets Zolgensma, told CNN.

"As a single, one-time intravenous infusion, Zolgensma's price in the countries where it has been approved reflects the long-term value it brings.

While this may be one of Lucy's only chances to live past two years, Van Doormaal said the cost of Zolgensma is overwhelming, and without help they simply will not be able to pay for it.

"They have put a price tag on my daughter's life, and that's extremely difficult to digest," she said.

While Lucy's parents fight the clock to raise the money needed to buy Zolgensma before her symptoms worsen, for now Lucy is being given Spinraza, a medication that slows the progression of SMA.

Spinraza usually costs approximately $750,000 for the first year and $375,000 for subsequent years for the rest of the patient's life, but the medication is completely covered by Lucy's health insurance in Canada, Van Doormaal said.

Until Lucy receives Zolgensma, if she is able to, her family and neurologist will not know for sure if she will need to continue using Spinraza for life.

Since SMA results in severe respiratory issues, Lucy sleeps hooked up to a BiPAP (Bilevel Positive Airway Pressure) machine, a non-invasive ventilator, to help her breathe.

Until they receive a potentially lifesaving gene therapy treatment like Zolgensma, their respiratory issues and muscle weakness will only aggravate.

"It's critical she gets the (treatment) as soon as possible," Van Doormaal said.

About 450 to 500 infants in the United States per year are born with SMA, according to Novartis, the parent company of AveXis.

The families of other babies, such as 1-year-old Kaysen Martin from Canada, are also racing the clock to raise money to acquire the gene therapy treatment for their children before it's too late.

While every child responds differently to Zolgensma, there is a chance Lucy will be able to outgrow the gastrostomy tube if she receives the treatment fast enough.

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