Vancouver baby with rare genetic disorder randomly selected for treatment program

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VANCOUVER (NEWS 1130) — There has been another positive development in the live-saving efforts for a Vancouver baby diagnosed with a rare genetic disorder.

Not only did the family of baby Lucy Van Doormal meet a fundraising goal to treat her Spinal Muscular Atrophy (SMA) type 1, she has now been randomly selected by a drug company for managed access treatment.

The initial goal of a fundraiser was to get the four-month-old a treatment of Zolgensma, a drug that isn’t available in Canada and costs about $3 million.

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But now that Lucy has been chosen for a treatment program through AveXis, the family is in shock.

“This program is a worldwide lottery and although, like other parents we entered Lucy into it, we never expected her to be selected,” Scott Van Doormaal writes on Lucy’s gofundme page.

For now, Van Doormaal says the family isn’t sure what they will do with the funds, but will need to wait until Lucy’s treatment to decide.

“That being said our goal and intention with the funds is to support the SMA community.”

The type of SMA Lucy has been diagnosed with is incredibly aggressive and if left untreated, she wasn’t likely to live to see her second birthday. The disorder attacks Lucy’s motor neurons and reduces her ability to swallow, sit, crawl, and control her head and neck.

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“We truly believe Lucy would not be receiving this treatment without the amazing support from all of you. Every personal story, kind message and fundraising effort gave us hope and strength to keep fighting for Lucy,” Scott Van Doormaa says. “We are so thankful for the awareness this has brought to SMA and are dedicated to continue to advocate for this community.”

AveXis is owned by the pharmaceutical company Novartis, which offers managed access programs to give certain treatments that are “investigational or unapproved” to eligible patients.

-With files from Renee Bernard

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