Canadian girl's medication for rare disease not completely covered by health care system

Renee Stocks, a four-year-old Canadian girl from a suburb of Ottawa, was diagnosed with the medical disorder 'Glutaric Acidemia type II', also called GA II, only two-and-a-half years after the first clinical signs of her illness appeared.

The very rare disease prevents the human body from producing enzymes needed to breaking down dietary fats and proteins into forms than can easily be handled by the body.
Without these enzymes, fats and proteins build up and will cause damage to the brain, liver, lungs and kidneys.

Had Renee been tested at birth, she could have been put on a strict diet that would have reduced her chances of serious health problems later in life, according to Frank Frerman, a professor of pediatrics at the University of Colorado Health Sciences Center.

Her parents had no idea anything was wrong with her until she became seriously ill at 9 1/2 months.
She was rushed to the hospital by ambulance after vomiting and a high fever that left her slumped over in her baby seat and then spent 72 days in intensive care on life support.

“We were passed from medical service to medical service, trying to find out what was wrong with her,” Renee's mother, Ms. Stocks said.

On Renee's third birthday, following a series of tests, including organ and muscle biopsies, the family got the bad news when a hospital in Colorado confirmed that Renee had GA II.

Renee's language development was behind her peers but she has since caught up with the help of speech therapy. However, she is taught at home instead of attending junior kindergarten classes at school, because the risk of coming in contact with a sick child is too high.

The Stocks have had to refinance the family home to help pay for Renee's special diet and put other plans on hold, including a summer vacation and finishing the basement.
The high carbohydrate diet prepared by the Hospital for Sick Children in Toronto includes fake chicken fingers and macaroni and cheese.

To restrict the amount of amino acids, which Renee cannot break down, the diet adds components of protein individually.
Ms. Stocks said the federal government recently agreed to pick up the tab for the diet.

But her big worry is how the family will pay for the experimental drug Renee started taking on December 1st.
The drug, known as L-3-Hydroxybutyrate, was developed by a Belgian doctor, Johan Van Hove, who now works at the Colorado lab, and costs $200 a day.

It is known as an orphan drug because the cost is not covered by private insurance plans or the Ontario government.
It is also well beyond the reach of what her husband, Andrew, earns as a paramedic.
Local residents recently held a fund raiser for the family, raising $14,200, enough for a two-month supply of the drug.

“I'm not bitter at the government for not funding this because I understand that there has to be checks and balances in place,” Ms. Stocks said. “But it is our only hope.”

Source: Globe and Mail

Editor's opinion:

"So, why exactly is this not covered by our system, why is she not urgent?
Why does someone that smokes (by choice) and gets lung cancer, receive proper and medical help covered by the system and the parents of this innocent little girl, without ever getting a choice of being healthy, have to carry the heavy financial load for treatment of their daughter??
ANGER and DISBELIEF, are the first words that come to mind.........."