Health Canada has approved Zolgensma, which is bringing renewed hope to Alberta families that have been tirelessly fundraising to afford its staggering cost of $2.8 million.
At least four children in Alberta have been diagnosed with the rare and debilitating disease spinal muscular atrophy (SMA).
According to Cure SMA Canada, SMA is a rare disorder that affects about one in 6,000 babies and causes muscles to waste away.
Zolgensma is a one-time dose gene therapy that is approved for SMA and would replace the faulty gene at the root of the disorder.
Two of the families recently received the treatment — Kaysen Martin through fundraising and Harper Hanki through a lottery run by a pharmaceutical company.
Bryarly Parker is the mom of Max Sych, who turns two in January.
Max was diagnosed in late November and the family had been racing against time to get him the treatment.
“You want this in him as soon as possible,” Parker said.
“You can see the loss of his motor skills every day.”
Reign Johnston, who turns two in March, is also waiting for treatment.
Parker said the SMA families keep in touch and provide support to one another.
“We are just in a community. It’s a group you never want to be a part of… but you’re so thankful to have them in every way.”
Parker said they are now in early talks with the provincial government and working with their MLA to get funding in Alberta.
“That’s our next step here,” Parker said. “It’s been a great day and we are lucky, but we still aren’t finished. and newborn screening for SMA is huge for us.
“It doesn’t end here.”
Alberta government spokesperson Zoë Cooper told Global News Wednesday that the provinces are now working together to negotiate a price for Zolgensma. Cooper said the province is “committed to providing access to children in Alberta who are eligible for this treatment.”
Minister of health Tyler Shandro’s press secretary Steve Buick told Global News the minister hopes to see Zolgensma funded early in the New Year.
Max was diagnosed in late November. Between the diagnosis, overwhelming GoFundMe support and Health Canada’s approval, Parker is still processing the past few weeks.
“We haven’t even had time to grieve ,” Parker said.
“This has been a rollercoaster of emotions, of news of what comes next. There is no path.
“It’s incredible what people will do to help you and to help your child. That has made me cry more than anything else.”
The current treatment option available for kids in Alberta is Spinraza, which must be administered in hospital and costs $375,000 a year. The prescription drug could be given for a child’s entire life and increases survival and motor function.
“He’s a strong little kid and he’s so happy. We are just so lucky to have Max,” Parker said.
“We just want what’s best for our little guy and this is that next step.”
© 2020 Global News, a division of Corus Entertainment Inc.