Pauline Kerr photo
HOPE FOR THE FUTURE – Zane and Lucas Braun have been participating in a trial for a new drug treatment that promises to significantly improve their quality of life. Both are on the drug and their mother Debbie Braun has seen a marked improvement in the boys’ health.
When Debbie Braun’s sons, Lucas, 16 and Zane, 14, were first diagnosed with Morquio A syndrome (also known as Mucopolysaccharidosis Type IVA or MPS IVA), a genetic metabolic disorder, the news was devastating. She was told there was no cure or treatment for the disease, and that they most likely wouldn’t live past their 20s.
Symptoms of Morquio A substantially limit both the quality and length of life of those affected, and may include systemic skeletal dysplasia, malformation of the chest, short stature, joint abnormalities, hearing loss and heart disease.
Lucas and Zane’s breathing and mobility have been impacted dramatically due to skeletal abnormalities and deformities caused by the disease.
Fortunately, the first-ever investigational treatment for Morquio A has been submitted to Health Canada under Priority Review. Vimizim has been shown to slow the progression of the disease, and the potential approval would fill a serious unmet medical need for Canadian patients.
Results of clinical trials demonstrate that Vimizim offers a substantial benefit to patients with Morquio A, and Lucas and Zane are already experiencing improved alertness, breathing, mobility and reduced need for orthopedic surgeries.
The approval of Vimizim is extremely important to Debbie and her boys, as it would not only make it the first available drug treatment option for Canadians living with this devastating disease; it would also mean that Debbie, Lucas and Zane could shift their perspective to focus on the future – something they could never have imagined before.
The drug’s manufacturer BioMarin Pharmaceutical Inc. announced last week that it submitted the drug under Priority Review status. The drug is an investigational enzyme replacement therapy for the treatment of patients with the lysosomal storage disorder Morquio A syndrome. Approval of Vimizim would make it the first available drug treatment option for Canadians living with this metabolic disorder.
“Morquio A is an extremely rare disease that substantially limits both the quality and length of life for those affected by it. There is no cure, and current available treatments are primarily symptomatic,” said Dr. Julian Raiman, a leading pediatric metabolic geneticist based in Toronto. “The potential approval of Vimizim would fill a critical unmet need for patients living with this severely debilitating, life-limiting and progressive disease.”
Health Canada designates Priority Review to drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists. Priority is intended to accelerate the regulatory review.
Debbie Braun said Lucas and Zane must travel each week to Toronto for the drug treatment.