Sask. will pay for a nearly $3 million therapy described as a game\u002Dchanger for newborns with a rare muscular disorder.
Those are the heartbreaking choices some families with spinal muscular atrophy have to make in order to access advanced gene therapies. One costs nearly $3 million, making it among the most expensive treatments in the world.
Desiree Parisien, a Saskatchewan resident with Cure SMA Canada, began taking Spinraza about two and a half years ago. She hoped the medication, administered four times a year via a spinal tube, would slow the progression of the disease so she could spend time with her husband and child, she said.The results exceeded expectations. Parisien said she regained muscle function she had been losing since she was a young girl. She was able to roll over, move to her knees and sit up without help.
They come at a steep price. Saskatchewan estimates roughly one in 10,000 newborns has SMA. The small size of the patient group, the advanced nature of the therapy and the massive cost of clinical trials means costs are sky-high.