Guelph family now in B.C. gets life-saving treatment for daughter, hopes to help others with funds raised
KITCHENER -- A Guelph family now living in B.C. has been given a possibly life-saving opportunity for their young daughter.
Four-and-a-half-month-old Lucy has a rare genetic disorder and needs a multi-million dollar drug.
The family was raising money for Lucy's treatment, but won't need the funds any more after the drug company selected Lucy to get the drug for free.
"This is giving us hope that she might be able to live a normal life expectancy," Lucy's mother Laura van Doormaal said.
She has a rare neuromuscular disorder called spinal muscular atrophy (SMA).
"It affects all of her muscles and that ranges from the ability to hold her head up or sit or even move her arms, to the really critical muscles of being able to swallow effectively and feed and also to be able to breathe," Laura said.
A life-saving drug, called Zolgensma, isn't available in Canada and has a $3 million price tag.
"It's the most expensive drug in the world right now," Laura said.
A GoFundMe campaign raised more than $2.4 million in just four months.
"From donations of almost 50,000 different people, and GoFundMe told us those people came from over 80 countries," Lucy's grandfather Brian van Doormaal said.
Two weeks ago, the company that makes Zolgensma said Lucy was randomly selected from a pool of candidates from around the world to get the drug for free.
"Lucy is going to get what we've been praying for," Brian said.
The company that makes Zolgensma said it launched a Managed Access Program (MAP) in January to make up to 100 doses of the drug available to eligible children free of charge.
In an emailed statement to CTV Kitchener, the company said choosing a recipient was based on fairness, clinical need and global accessibility.
A physician was able to submit a child into the MAP.
The family is left with millions of dollars from the fundraiser and said they plan to use the majority of those funds to support the SMA community.
"There are six other babies in Canada that we know of that are also trying to raise the funds for this treatment," Laura said. "We would love to be able to help them."
A spokesperson for GoFundMe said the company is working closely with the family to make sure the money will "go towards supporting the SMA community and remain in the spirit of the initial fundraising goal."
The statement also said the family will use some of the money to cover other costs related to Lucy's treatment.
"What's most important is that the donors' intentions are honoured and we will work with the family to let donors know how funds raised will be used," the statement said in part.
Donors are able to request a refund, GoFundMe said.
Lucy is expected to get her treatment in September.
"We know for sure with Zolgensma, she will have a longer life," Brian said. "Saving her life is our goal, and we feel we have done that."