Posted: Aug 8, 2012 8:57 PM CST
Last Updated: Aug 8, 2012 9:15 PM CST
A youngster with a rare medical condition has been approved for coverage of a costly drug, the only known therapy for MPS VI, also known as Maroteaux-Lamy Syndrome.
Patients with the condition lack a key enzyme in their systems.
A therapy is available but costs around $250,000 to $300,000 per year for a patient.
The family of 3-year-old Violet Revet recently appealed to provincial politicians to help them through the approval process.
A decision, which was announced Wednesday, means the treatment involving infusions of a replacement enzyme can begin.
According to officials, only nine people in Canada have the disease.
Revet’s situation caught the attention of a national foundation which mounted an awareness campaign, using social media such as Facebook and Twitter, to encourage providing coverage of the treatment.
On Wednesday, the Isaac Foundation said news of the approval was welcomed by the girl’s family.
“Its a relief to finally get the ball rolling on her treatments after waiting seven long months,” Larry Revet, Violet’s father, said in a news release from the foundation. “We can now move ahead with the next step to ensure Violet has a stronger, healthier life ahead of her. This is great news.”
“My daughter has been given a chance for a bright future,” Jennifer Revet, the youngster’s mother, added. “For this I am truly thankful.”
The foundation’s director, Andrew McFadyen, noted the approval process highlights the need for Canada to improve policies related to what are known as orphan drugs, or medications for extremely rare conditions.
“Families dealing with rare disease shouldn’t have to have put their children on the front page of newspapers to receive the treatment they deserve,” he said.