You may recall my previous posts about the Isaac Foundation. Good to see the government come through.
From the Globe and Mail:
The Ontario government has decided to fund a staggeringly expensive drug for Isaac McFadyen, the toddler whose parents were planning to move to England where the only treatment for his rare and progressive disease is funded.
Health Minister George Smitherman telephoned the boy's father, Andrew McFadyen, yesterday to say the province has decided to fund enzyme replacement therapy to treat the two-year-old boy's condition, known as Maroteaux-Lamy syndrome. The drug, Naglazyme, costs $300,000 to $1-million annually.
"This is life-changing for Isaac," Mr. McFadyen said yesterday. "It's going to provide him with potentially a very normal quality of life; it's going to halt the disease, and at best, it's going to reverse all the symptoms he is currently displaying."
Yesterday, David Spencer, spokesman for Mr. Smitherman, said the drug was accepted for funding after a clinical review was completed by members of Ontario's inherited metabolic diseases program. He said the Health Ministry was in a position to fund the drug only after this review.
"We have to make sure any decision is based on good science and a clinical recommendation," Mr. Spencer said yesterday. "That was possible in this case. . . . It's always a pleasure when we can come to this result."
People like Isaac are missing an enzyme needed to break down carbohydrates, which build up in the body's cells and affect many organs.
Signs of the disease include stunted growth, enlarged tonsils and adenoids that cause breathing problems, poor mobility and dramatic changes in facial features, including a flat nose and large head.
Once sufferers reach their teens, they often require heart-valve surgery. An estimated three to 10 Canadians suffer from the metabolic disorder, known as MPS VI.
The boy's plight was described in a Globe and Mail story that examined the absence of an orphan-drug policy in Canada to cover the high drug costs associated with rare diseases.
At the time, Mr. McFadyen, an elementary school teacher in Kingston, and his wife, Ellen Buck-McFadyen, a public-health nurse, felt they had no choice but to look for employment overseas to get treatment for their son.
But all that has changed. The couple now plan to stay in their dream home in Campbellford, Ont., and commute to Toronto's Hospital for Sick Children, where Isaac will receive treatment.
"This changes the course of his future," Mr. McFadyen said. "It's incredible, I can't believe it."