Children suffering from crippling genetic disease
CBC News Posted: Oct 05, 2015 6:43 PM CT Last Updated: Oct 05, 2015 7:00 PM CT
The province’s health ministry says it won’t pay for a costly drug that may help three children with a crippling genetic disease, but Health Minister Dustin Duncan said he will ask for a second opinion.
Muhammad Abdullah, 12, Khadija Amir, 10 and Sara Amir, 8 all have a rare enzyme deficiency called Morquio A Syndrome (also called MPS IV type A).
Without the missing enzyme, cellular waste builds up in the bones, tissues, organs and muscles.
The condition is stunting the children’s growth, twisting their joints, affecting their eyesight and hearing, and making it tough for them to breathe. Two of them use wheelchairs at school.
Muhammad Amir Akhter is the children’s father. He said the disease can be life-threatening.
“Enzyme replacement therapy like Vimizim is the last resort,” Akhter said.
He said despite the current decision, he is staying positive. “It’s pretty hard for us, because this is the last hope here.”
Health minister will get a second opinion
Health Minister Dustin Duncan explained the decision to reject the drug coverage.
“These are very, very complex cases,” said Duncan.
He said in this case there is not enough evidence to prove that the drug will be effective for people over the age of five.
“There’s a lot of opinions on this. And so while we do rely and have relied on a previous case, on an out-of-province expert I’m asking the ministry to consult with others that may have some information on this particular case.”
Duncan said the ministry will review some of the medical debate about the efficacy of the drug, and will invite the Akhter family to submit any further evidence they find.
“While the answer last week was no, it doesn’t mean it’s no forever.”
The Opposition NDP said the children should get the drug since it is their only hope. It added that effectiveness can be monitored while they take it.