A B.C. family who raised almost $3 million for a drug to help their 4.5 month-old baby girl with spinal muscular atrophy (SMA) has now received some more good news.
The drug company Novartis/AveXis, which makes the very expensive drug Zolgensma, gifts a treatment to someone about every two weeks.
Little Lucy Van Doormaal was chosen from a worldwide pool of candidates.
“We were just in complete shock and then obviously overjoyed,” mom Laura told Global News.
Zolgensma will give Lucy the gene she is missing, and although it won’t correct any loss of muscle movement so far, it will stop the spread of SMA.
“It seems surreal,” Laura said.
“We are eternally grateful to the community that came out to support Lucy. It has been extremely overwhelming but also very uplifting as well and helped to create some more awareness of SMA, so that’s been a great side effect of our campaign.”
The fundraising campaign has now been closed but the Van Doormaals are not sure what they are going to do with the money raised for Lucy to get the treatment.
“It would be our intention and goal to use that money to support the SMA community,” said Laura.
“There’s a lot of need in the SMA community and specifically for children searching for the drug Zolgensma so we want to have the biggest impact we can.”
Lucy still has a lot of limitations but Laura says physiotherapy has been helping and hopefully the drug will help her begin to make bigger improvements.
She will now undergo more testing to makes sure she is still a good candidate for the treatment. If it goes ahead, it will be the first time it will be performed at B.C. Children’s Hospital.
Lucy will have to be given a steroid following the dose to make sure her body doesn’t fight off the virus that is giving her the new gene.
“We think it’s the best chance she has to living a long and healthy life with SMA. But it’s not a cure. She’ll always have SMA,” Laura said.
“We hope the best for Lucy and we’re thankful to have support in this journey but we’re still processing the realities of our new life.”
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