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GoFundMe aims to get Alberta toddlers with rare genetic disease a stem cell transplant

An Okotoks family is hopeful that first-of-its-kind stem cell therapy can help after two young children were diagnosed with an extremely rare genetic disorder
The Beet family.

An Okotoks family is hopeful that stem cell therapy can help two young children diagnosed with an extremely rare genetic disorder called Morquio Syndrome. 

Otherwise known as Mucopolysaccharidosis Type IV (MPS IV), the disease causes waste sugars in the body to progressively build up, leading to a range of complications. It is estimated to occur once per 200,000 to 300,000 births. 

The family posted a video on YouTube where it talked about what it has been going through since the diagnosis. 

Four-year-old Stella was diagnosed first, following what the family thought would be a routine doctor’s appointment, said Stella’s mother, Brittny Beet.

Within six weeks, younger brother Max was also found to have the disease, Beet said. 

“Because it's genetic, we had a one-in-four chance of any of our children getting this disease,” she said. 

Morquio Syndrome causes multi-systemic problems and bone deformities, leaving up to 70 per cent of patients in a wheelchair by their teen years. 

The life expectancy for patients is between 30 and 40 years with intervention, Beet said. 

“Having a life expectancy put on your children is a nightmare I wish on no parent,” she said. 

Once the diagnosis sunk in, the family got to work and started reaching out to anyone who might have information about the disease and possible treatment. 

Alberta Children’s Hospital discussed the case and doctors agreed that stem cell therapy was an option, Beet said.

Stem cell donors were found for both kids and transplants will start after a round of chemotherapy to ensure they do not reject the transplant. 

Max’s treatment will begin once Stella’s is complete. 

The hope is the therapy will help the children produce the enzyme needed to break down waste sugars on their own, she said.

“They'll be the first children in Canada to have it done for their disorder,” Beet said. 

With a long road ahead, a GoFundMe campaign has been started to assist the family with some of its expenses. 

“We're very hopeful for what's to come,” Beet said. “We have faith in Alberta Children's Hospital and the treatments that we're about to go through with the kids.” 

Robert Korotyszyn

About the Author: Robert Korotyszyn

Robert Korotyszyn covers Okotoks and Foothills County news for and the Western Wheel newspaper. For story tips contact [email protected]
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