I stood on the edge of a small Manitoba community hall, watching as volunteers hung blue and white streamers from the rafters. In the corner, a donation box overflowed with checks and cash – each contribution representing not just money, but a collective promise to seven-year-old Liam Johnston.
“We didn’t expect this many people to show up,” whispered Sarah Johnston, Liam’s mother, as she surveyed the growing crowd. Her eyes, rimmed with exhaustion but bright with hope, kept darting to the front door. “It feels like the whole town is here.”
The whole town of Minnedosa, Manitoba – population just over 2,500 – had indeed shown up, along with families from neighboring communities. They gathered last weekend not for a hockey game or harvest festival, but to help the Johnston family access a life-changing medication for Liam that costs $2.8 million for a single dose.
Liam was diagnosed with spinal muscular atrophy (SMA) type 3 when he was four. SMA is a rare genetic disorder affecting approximately one in 10,000 people that causes progressive muscle weakness. For Liam, this means struggling with activities other children take for granted – climbing stairs, running at recess, even maintaining his balance while standing.
“He falls a lot,” Sarah explained. “Each time, it gets harder for him to get back up.”
The medication that could change Liam’s life is Zolgensma, a gene therapy treatment approved by Health Canada in 2020. It works by providing a new copy of the gene that makes the protein critical for motor neuron survival. Clinical trials have shown remarkable results in young children, potentially halting disease progression and even restoring some motor function.
But the astronomical price tag places it among the world’s most expensive medications. While Manitoba Health covers Zolgensma for children with SMA type 1 – the most severe form – Liam’s type 3 diagnosis falls outside current coverage guidelines.
“We applied for exceptional access three times,” said Mark Johnston, Liam’s father, as he helped arrange silent auction items donated by local businesses. “Each time we were denied because his condition isn’t considered severe enough yet. But the research shows the earlier kids get treated, the better the outcomes.”
The fundraiser represented more than just financial support – it embodied a community’s refusal to accept bureaucratic boundaries when a child’s future hangs in the balance.
Dr. Hanns Lochmüller, a neurologist and researcher at the Children’s Hospital of Eastern Ontario, has seen firsthand how access to treatment varies dramatically across Canada’s healthcare landscape.
“For rare diseases, we have a patchwork approach to coverage,” Dr. Lochmüller said when reached by phone. “Some provinces cover certain treatments while others don’t, creating significant inequities for Canadian families depending on where they live.”
This disparity is particularly stark for expensive treatments like Zolgensma, which provincial health authorities evaluate independently. While Ontario recently expanded coverage to include some SMA type 2 and 3 patients under age two, Manitoba’s criteria remain more restrictive.
At the community hall, I watched Liam navigate through clusters of neighbors and friends. Despite his unsteady gait, he moved with determination, his “Future Scientist” t-shirt reflecting his passion for experiments and discovery.
“He doesn’t want special treatment,” Sarah noted. “He just wants to keep up with his friends.”
The Johnston family’s struggle highlights the broader challenges facing the estimated 3 million Canadians with rare diseases. Last year, the federal government promised a national rare disease drug strategy with $1.5 billion in funding over three years starting in 2025-26. For families like the Johnstons, however, such promises offer little immediate relief.
Local teacher Emma Winthrop organized a school fundraiser that brought in over $3,000 toward Liam’s treatment.
“Kids understand fairness intuitively,” Winthrop said. “When we explained that Liam needs medicine that costs more than most houses, they immediately wanted to help. One second-grader even donated her entire piggy bank.”
The community’s efforts have raised nearly $400,000 so far – an extraordinary sum for a small rural community, but still far short of what’s needed. The Johnstons have also launched an online campaign that has attracted donations from across Canada.
Dr. Alex MacKenzie, a pediatrician and researcher at the CHEO Research Institute who specializes in rare genetic disorders, points to the fundamental dilemma these situations create.
“These are truly remarkable treatments that can transform lives,” Dr. MacKenzie explained. “But our healthcare system wasn’t designed to accommodate medications with such extreme costs. We need new models that balance innovation, access, and sustainability.”
As evening fell on the fundraiser, volunteers counted the day’s proceeds while Liam demonstrated his science experiment – a homemade volcano that erupted with baking soda and vinegar to delighted applause.
“A lot of people ask why we don’t just move to Ontario where he might qualify for coverage,” Mark said. “But this is our home. Our support system is here. Today proves that.”
Health policy experts like Steve Morgan, a professor at the University of British Columbia’s School of Population and Public Health, suggest that rare disease treatments expose fundamental gaps in Canada’s healthcare framework.
“Our system excels at hospital care and physician services,” Professor Morgan noted, “but pharmaceuticals have always occupied this strange middle ground where coverage varies wildly. For rare diseases, this creates devastating inequities.”
The evening concluded with Liam’s grandmother reading his favorite story to a circle of children seated on the community hall floor. As I prepared to leave, Sarah Johnston touched my arm.
“People sometimes ask if we’re angry about having to fundraise for healthcare in Canada,” she said quietly. “The truth is, we’re too busy being grateful for this community to be angry. But yes, the system needs to change – not just for Liam, but for every child who needs help.”
As Manitoba’s endless prairie sky darkened outside, the Johnston family’s journey illuminated something essential about both the strengths and limitations of Canadian healthcare – a system built on principles of universality that sometimes fails its most vulnerable citizens, leaving communities to fill the gaps with bake sales, silent auctions, and an unwavering belief that every child deserves a fighting chance.
For more information on spinal muscular atrophy and patient support resources, visit Cure SMA Canada or the Neuromuscular Disease Network for Canada.
