Violet Revet, a three-year-old girl from Langenburg, is one of only nine people in Canada who has Mucopolysaccharidosis VI (MPS-VI) and now she will receive treatment she desperately needs.
MSP-VI has no cure and the longer she goes without treatment, the shorter she could live.
Andrew McFayden’s son, Isaac, was diagnosed with the disease in 2005.
“It’s a very devastating condition. Essentially kids that are diagnosed with MPS lack an enzyme in their blood that breaks down all the complex sugars. Because those complex sugars that occur naturally in the body don’t break down they need to find a place to go and so they end up being stored in the bones, tissues, organs, and muscles and it leads to many progressive symptoms: heart and airway disease, progressive stiffening of the joints, corneal clouding, spinal cord compression.”
“Essentially every muscle, tissue, and organ in the body is affected by this disease with the exception of the brain.”
McFayden was told Isaac would only have seven or eight years to live. That was before doctors realized a treatment was making the rounds in the UK and the US, a synthetic enzyme known as naglazyme that acts as a replacement in the body.
Isaac could get access to it under a special access program but the drug wasn’t covered in Ontario. It took McFayden eight months of lobbying before the provincial government agreed to pay for it.
Violet’s family asked Saskatchewan’s health ministry for funding so she could get the same treatments.
Today, the government drug review process has approved naglazyme and her doctor will begin administering the treatment.
With files from News Talk Radio’s Patrick Book