The B.C. baby who made headlines around the world after it was revealed she needed a multi-million dollar drug to fight spinal muscular atrophy, also known as SMA, has finally received a dose.
Lucy Van Doormaal received a dose of Zolgensma at B.C. Children’s Hospital on Sept. 9.
“It was a day charged with emotions as we finally took a deep breath of relief,” her parents wrote on their GoFundMe page.
The family had started the fund with the goal of raising $3 million for Zolgensma, a drug often described as the most expensive in the world.
However, it was revealed in mid-August that the drug company that makes it, Novartis/AveXis, had gifted it to the Van Doormaals from a worldwide pool of candidates.
It is a one-time dose, and although it is not a cure for SMA, it will stop the progression of the disease.
“She will always have SMA and she will always live with a disability but hopefully now she will have the best chance to have a healthy life,” Lucy’s parents wrote Thursday.
They raised more than $2.4 million before they were told Novartis/AveXis was gifting them the drug, so they had to decide what they were going to do with all the money raised.
In the update posted online, they said they will donate “a large portion of the funds directly to other Canadian SMA1 babies to help them reach their Zolgensma goal.” They will also donate money to Muscular Dystrophy Canada and the B.C. Children’s Foundation and keep some of the funds for Lucy’s future medical needs.
In the final GoFundMe update, her parents said she handled the infusion well and she is now at home recovering.
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