9 October, 2015 mcfadyena

Medical treatment funding plea for children with rare genetic disease


SASKATOON – Amir Akhter is waiting for a life-changing phone call, a phone call that could determine the future of three of his children. Earlier this week, the Saskatchewan government denied funding an expensive treatment that may help them live longer.

The three children suffer from the same rare genetic disease.

“Skeletal disorder, hearing loss, vision loss, spinal cord problems, their heart, lungs, almost every part of the body,” said Akhter.

READ MORE: Rare genetic disease diagnosed in 3 kids from same Saskatoon family

But a treatment called Vimizim could change things. It costs hundreds of thousands of dollars and the Akhter’s were hoping the province could foot the bill.

That was rejected on Monday by the Saskatchewan government.

“The experts at this point have said there really doesn’t appear to be efficacy when it comes to halting the progression of the disease as they get older, but I want to have a second look at it,” Health Minister Dustin Duncan said at the time.

He added that because the kids are past the age of five, the treatment might not even work.

READ MORE: Treatment funding denied for 3 children with rare genetic disease

The Opposition NDP says that’s not true at all.

“That is the exact opposite of what the evidence says,” said Danielle Chartier, the NDP health critic.

“You can look at Vimizim’s own fact sheet that proves the drug is effective in those older than five.”

Chartier added that another child in the province is getting the same kind of treatment. Her age has not been confirmed.

For Akhter, it’s not about the back and forth disputes, it’s about watching his children deteriorate every single day.

“It’s heartbreaking, but we are not losing our heart, we are still very positive,” said Akhter.

“Hopefully, my kids will be getting that treatment.”

Biomarin Pharmaceuticals, a manufacturer of Vimizim, confirmed that safety and effectiveness have been proven for kids five year or older on their website.

The Health Canada website too says it is the other way around, that efficacy hasn’t been established for those under five.

The family is waiting to hear back about a clarification and a final decision.

Leena Latafat contributed to this story

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