Kids with disorder given ‘new life’
Saskatchewan agrees to cover treatment for siblings with ‘ultra-rare’ genetic condition
Every night, Amir Akhter faced questions from his son that most people don’t have to think about until the end of their lives.
Muhammad Abdullah is just12, and his body is closer to that of a 4-yearold.
“All the time, they have many questions for us and we have no answers, in fact,” Akhter said by phone Thursday from Saskatoon. Muhammad Abdullah and two of his four siblings have an “ultra-rare” genetic condition known as Morquio syndrome, which affects less than 0.1 per cent of the population. It stunts their development, affecting every part of their body except the brain.
Muhammad and his two sisters, Khadija, 10, and Sara, 8, are now in wheelchairs while his eyesight is rapidly declining. Until this week, their lifespan wasn’t more than 30 years.
“I tell him, ‘No worries, you don’t need to worry about that.’ But frankly speaking, when he gets to sleep, we are thinking about the future and their life. We have nothing except years,” Akhter said.
The couple also has a 16-year-old daughter, Shafaq, and a 5-year-old son, Abdul Salam, who do not have the disease.
There is no cure for the syndrome, but a new enzyme-replacement drug called Vimizim — which was intended for palliative use before it was approved for the market last year — has revolutionized treatment.
Tuesday, the Akhters learned the government of Saskatchewan would be covering the treatment, worth more than $200,000 a year per child. The decision, following an external review, reverses the provincial government’s previous refusal earlier this month.
“After hearing the challenges faced by this family, I asked the Ministry of Health to revisit the initial decision,” Saskatchewan Health Minister Dustin Duncan said in a Nov. 3 press release. “Any further decision to provide coverage for Vimizim will be made on a case-by-case basis.” Andrew McFadyen, who founded the Isaac Foundation after his son was diagnosed with another type of the disease 10 years ago, says Saskatchewan’s decision sets a precedent in Canada because of the expert review process that reversed the decision. For example, only one child was approved coverage in Ontario and the other eight patients in the province receive the treatment courtesy of the drug company.
“This is, in fact, a new life, a new sun shining for our life,” Akhter said of the news he received.
“They have very big plans for their future,” the father said of his children. Muhammad asks about driving when he turns 16; he wants to play soccer, too.
Vimizim hasn’t been around long enough to determine by how long it can prolong the lives of those with the syndrome, but the treatment effectively helps them live a normal life, McFadyen said.
As far as Akhter concerned, his children now have as many chances of surviving as anyone else in the world. “Everything is finite.”