Published:
FOR IMMEDIATE RELEASE: The Minister of Health in Saskatchewan has approved funding for a life-saving treatment required by three siblings from Saskatoon. 8 year-old Sara Amir, along with her siblings Khadija, 10 and Muhammad, 12, suffer from MPS IVA (Morquio Syndrome) and require the life-saving treatment immediately in order to halt further progression of their devastating disease. After initially denying funding for the children, the Minister of Health ordered a review of the files and asked a panel of experts to provide feedback to the Ministry regarding the children and the treatment required.
Sufferers of Morquio Syndrome lack an enzyme in their blood that breaks down cellular waste in the body. This waste builds up in the bones, tissues, organs, and muscles of affected individuals and lead to many devastating symptoms including heart and airway disease, corneal clouding, stiffening of the joints, shortened stature, and premature death.
Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, was thrilled with the news. “I applaud the Minister for his leadership during this review, and thank him for the open and transparent way with which he conducted this review. This is life-saving for these children, and life-changing for their family. This is incredible news.”
Jamie Myrah, Executive Director of The Canadian MPS Society, also thanked the NDP Health Critic, Danielle Chartier, for her work bringing this issue to the legislature and for the advocacy she provided the family during what was a very emotional period.
McFadyen adds, “The Saskatchewan government has taken a leadership role on this and have come in line with the Canadian Expert Opinion and the International Treatment Guidelines for Morquio Syndrome. They have also mirrored the recent recommendations from the UK. Most importantly, they’ve given hope back to this family – hope for a new life and the chance at fighting back against this relentless disease. For that, I am incredibly thankful to Minister Duncan and his team.”
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For more information about this topic, or to schedule an interview with Andrew McFadyen, please call Andrew at 613-328-9136 or email Andrew at mcfadyena@me.com. The Isaac Foundation can also arrange interviews with parents of patients currently receiving this treatment in Canada.
Backgrounder
While not a cure for Morquio Syndrome, the necessary Enzyme-Replacement Therapy (ERT) is designed to provide patients with a synthetic version of the enzyme they are lacking by infusing small doses into the patient’s bloodstream on a weekly basis. The treatment slows down or halts the progression of the disease in patients, improves endurance, walking distance, breathing problems, and provides other benefits to sufferers that dramatically improve their quality and length of life. International experts and a Canadian Panel of Genetics Specialists have all recommended Vimizim as the front-line treatment for Morquio Syndrome. It was approved by Health Canada in July 2014, and has been reimbursed for use by patients in Saskatchewan, Ontario, and Quebec. Recently, the National Institute for Health and Care Excellence (NICE) recommended reimbursement for all 88 patients suffering from Morquio Syndrome throughout the UK.