Treatment approved for St. Albert toddler with rare disease
By: Amy Crofts
| Posted: Monday, Aug 12, 2013 03:30 pm
After more than a month of lobbying the provincial government to fund a life-sustaining treatment for their toddler, Aleena Sadownyk’s family can breathe a sigh of relief.
The family received word Monday that the province has approved the enzyme replacement therapy used to treat the extremely rare Maroteaux-Lamy Syndrome, also called MPS VI.
“My daughter has been given the chance for a bright future … For this I am truly thankful,” said Aleena’s mother Laura Sadownyk in a press release by the Isaac Foundation. The organization’s mission is to find a cure for MPS as well as aid families in advocating for treatment funding.
Three-and-a-half-year-old Aleena was diagnosed in April. Due to the lack of the enzyme glycosaminoglycan, her body is unable to break down cellular waste. The waste then builds up in the muscles, organs and bones, causing stiff joints, corneal clouding and heart and airway disease, amongst other serious health complications.
The enzyme replacement therapy Naglazyme runs at a price of $300,000 to $1 million per year. Treatment has been funded in provinces including British Columbia, Saskatchewan, Ontario, and Quebec, but was denied by Alberta Health under the Alberta Rare Diseases Funding Program.
“The past few weeks have been very difficult for us, but we can now focus on improving Aleena's well-being and look forward to her having a bright future ahead,” said Dane Sadownyk, Aleena’s father.
Aleena will soon begin the weekly Naglazyme infusions. To date, there are nine children suffering from MPS in Canada and roughly 1,100 worldwide.