Published:
BY ANGELIQUE RODRIGUES ,EDMONTON SUN
FIRST POSTED: FRIDAY, AUGUST 02, 2013 07:14 PM MDT | UPDATED: FRIDAY, AUGUST 02, 2013 07:35 PM MDT
St. Albert Toddler Diagnosed With Ultra-Rare Condition; Treatment Already Being Funded In Numerous Provinces Alberta Health is currently considering an application for exceptional funding of an expensive life-sustaining treatment required by an Alberta toddler. Three year-old Aleena Sadownyk was recently diagnosed with MPS VI, and requires the life-sustaining treatment immediately in order to halt further progression of her devastating disease. Alberta Health has already denied funding for the treatment through the Alberta Rare Diseases Funding Program. They are now reviewing a second application through the Short Term Exceptional Drug Therapy (STEDT) program. While Aleena needs to begin treatment immediately, there has been no timeline set for a decision from Alberta Health. Family Hand Out Photo
A St. Albert toddler with a rare disease is fighting for her life while the province debates whether the treatment she needs should be covered by health care.
Three-year-old Aleena Sadownyk was recently diagnosed with a rare and fatal enzyme deficiency called Maroteaux-Lamy Syndrome or MPS VI.
The toddler is already experiencing serious symptoms — Aleena’s hands and shoulder joints have begun to seize up — and without treatment officials say the disease is likely a death sentence.
“Left untreated, children have a severely shortened life-span,” said family-friend Andrew McFayden.
“There are varying degrees of progression in children and right now it’s suspected that Aleena has a rapidly progressive form.”
McFayden is a new friend of Aleena’s parents — Laura and Dane Sadownyk.
The Sadownyk’s reached out to McFadyen after they learned about his son Isaac — who lives with the same disease their daughter was diagnosed with — and McFadyen’s fight to have the Ontario government foot his treatment bill.
Individuals with MPS VI require an enzyme replacement therapy that can cost up to $1 million per year, and the treatment must be continued once a week for the rest of their lives.
It’s already covered by provincial health care in Ontario — where McFadyen battled to have the treatment brought to Canada using the Federal Government’s Special Access Program (SAP) and paid for by the province, as well as in British Columbia, Saskatchewan and Quebec.
Now, McFadyen is hell-bent on helping Aleena get the treatment that has allowed his son — who was crippled with spinal pressure and enlarged internal organs — to burst through their back door today at a full run.
“As I’m talking to you right now he’s racing outside to play with his brothers,” said McFadyen, of Isaac, 9, who has been receiving the treatments for seven years now.
“The damage that was done to his spine, bones and joints can’t be reversed but there has been no further build up of disease.”
Though he’s grateful it wasn’t worse, he doesn’t want to see Aleena left with the same life-long damage.
“Aleena is waiting to begin treatment - her hands are beginning to claw up, she can’t lift her shoulders above her head, that’s the latest,” he said. “She needs to start treatment in order to halt any further deterioration.”
According to McFadyen — who runs a charity called the Isaac Foundation — Alberta Health has already denied funding for the treatment through the Alberta Rare Diseases Funding Program.
But officials are currently revising a second application through the Short Term Exceptional Drug Therapy (STEDT) program — with no timeline set for a decision.
It’s time Aleena doesn’t have, and McFadyen says his hope is for Premier Alison Redford to step up and expedite the process.
“The provincial government has the opportunity to do the right thing and ensure that Aleena receives the treatment she so desperately needs,” he said.
“Parents should not have to put their children’s faces on the front of newspapers in order to get the treatment they deserve.”