Parents raised $2 million for their baby's life-saving medical treatment, and then got the treatment for free

Parents raised $2 million for their baby's life-saving medical treatment, and then got the treatment for free
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  • Laura and Scott Van Doormaal spent months fundraising to afford a life-saving treatment for their daughter, Lucy.
  • Lucy has spinal muscular atrophy, and was not expected to live past two without treatment.
  • The Van Doormaal family lives in Canada, where the treatment is not yet approved, but was able to get access through a program offered by the drug maker.

Earlier this year, Laura and Scott Van Doormaal turned to the internet to help save their daughter's life, raising $2.5 million on GoFundMe for a drug infusion that could save her. But then luck intervened, and Lucy Van Doormaal, who was born with the rare genetic condition spinal muscular atrophy (SMA), got the treatment for free.

"In an incredible turn of events, we found out Lucy was randomly selected by the drug company, Novartis/AveXis, to receive treatment through the managed access program," the Van Doormaals shared on GoFundMe back in August.

The program is meant to help children in countries where the treatment — Zolgensma — isn't yet approved for use. That includes Canada, where the family lives. The program was just launched this year and will provide treatment to 100 children, the company told CNN.
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"While we aren't providing specific numbers at this time, we can confirm that already the program has enabled children across Asia, Australia, Europe and North America (including Canada) to receive treatment who may not have otherwise had access to the therapy," a Novartis Gene Therapies company spokesperson said.

Immediate results from a life-saving treatment

Lucy received the infusion on September 9 at British Columbia Children's Hospital in Vancouver, according to the family's GoFundMe campaign.

"It was a day charged with emotions as we finally took a deep breath of relief," her parents wrote on GoFundMe. Right away, they noticed some improvements in Lucy's muscle control.
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"We noticed an immediate difference," Laura Van Doormaal told CNN. "The biggest changes are her head control, really quickly after the treatment she was able to hold her head up which is something she's never done before."

Although it's not clear what the future holds for Lucy, that quick progress has left the Doormaal family hopeful. "She can sit with some support and hopefully one day she'll crawl," Van Doormaal said. "Maybe one day she'll even walk. That's really an optimistic dream, I know, but we like to have high expectations and we don't want to hold her back."
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Since Lucy received treatment for free, many people asked what would happen to the $2.5 million that the family raised from people around the world. In early October, the family posted to GoFundMe, explaining their intentions for the funds and allowing anyone to request a refund of their donation.

Although the update didn't go into specifics, it said that a "large portion" of the funds would be donated to seven other Canadian babies who have the same condition as Lucy. For at least one of the babies, Eva Batista, that will mean that she's able to access treatment.

Funds will also be donated to Muscular Dystrophy Canada and the hospital where Lucy received her infusion. Finally, some money will be set aside for "Lucy's future and complex medical needs."
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Now that treatment has been administered, the family is scaling back from GoFundMe to focus on Lucy's care, they wrote on Sept. 17.

"Our quest for Zolgensma was exhausting yet uplifting. In many ways it changed us as parents, bringing us to extremely dark places but also showing us the amazing strength of human kindness and community. We feel so fortunate to have reached our goal of providing this treatment for Lucy. You are all a huge part of this success."

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