Reign Johnston’s family met Kaysen Martin’s when both babies were in Edmonton’s Stollery Children’s Hospital. The boys have the same rare disorder and their families say they both need the same expensive treatment.
“We actually made a fairly substantial contribution to Kaysen’s campaign because we want to see him get Zolgensma,” Reign’s father, Ryan Fengstad, told Global News.
“We’re fighting it together with them.”
Both Edmonton boys have spinal muscular atrophy (SMA) type 1. The rare disorder affects about 1 in 6,000 babies, and causes muscles to waste away. Most die of respiratory issues before their second birthday.
Zolgensma, a new gene therapy, replaces the function of a child’s missing gene. Studies show 91 per cent of patients who received the one-time infusion were still alive at 14 and 18 months, with no need for permanent breathing support.
The therapy costs $2.8 million and is not yet approved in Canada. Reign’s parents have been raising money for him to go to the U.S. for treatment, but COVID-19 has made fundraising difficult and travel impossible.
Another barrier is that Zolgensma is only approved by the U.S. Food and Drug Administration for babies under age two because it is deemed too risky in older children.
That means even if Canadian families can be approved through Health Canada’s Special Access Program, the age limit would likely remain.
Time is running out for both Edmonton patients. Reign will turn two years old on March 5, 2021. Kaysen’s second birthday is on July 17, 2020.
The manufacturer of Zolgensma, Novartis Canada Inc., emailed a statement to Global News about the challenges posed to some Canadian families seeking the treatment.
Reign’s parents said they reached out to both Novartis and Alberta Health for help months ago, but have not heard anything since.
Tom McMillan, Alberta Health’s assistant director of communications, emailed Global News and said that if Health Canada does not approve the use of Zolgensma for babies older than two, the province will ask Novartis to consider providing access through a clinical trial.
Reign’s mother, Alex Johnston, wants the decision-makers to know her son “is worth it.”
Reign’s father dreams of a day when there are no barriers to life-saving treatment.