A Regina mother who was one of the first ALS patients to test a new treatment is now celebrating its accelerated approval in Canada for adults with an ultra-rare form of the disease.
Paula Trefiak’s remarkable experience on tofersen, a medication sold under the brand name Qalsody, is reflective of why researchers are calling the drug a breakthrough.
“My life has completely changed. I am actually looking forward to retirement now. I never thought I would ever get to retirement,” Trefiak, 43, told CBC News.
People diagnosed with amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disorder also known as Lou Gehrig’s disease, have a life expectancy of two to five years. They become trapped in their body, losing the ability to move, speak, chew and eventually breathe.
Health Canada confirms it has granted conditional approval to Qalsody to treat adults with ALS in cases linked to a mutation in the …