‘THE RIGHT PLACE’: Ottawa baby a pioneer of gene therapy for rare disease
Aidan Deschamps can crawl, climb stairs and, with the help of an ottoman or chair, stand. When his mother, Amanda Sully, or father, Adam Deschamps, holds his hands, he can even take some steps.
In other words, he’s cruising like most 10-month-old babies and hitting all the developmental milestones expected of him.
But things could have turned out much differently had Aidan been born a week earlier, or anywhere in Canada outside Ontario.
For, unbeknownst to him, the Ottawa baby is a pioneer in medicine, the first in Canada to be diagnosed with spinal muscular atrophy (SMA) using the province’s newborn screening test, and the first in the country, outside of clinical trials, to be subsequently treated for the rare and often fatal neuromuscular disease using gene replacement therapy.
“There are no words for it,” his father said in a press conference held Wednesday to provide an update on Aidan’s health. “We are just so blessed that we live in the right place and that Aidan was born when he was. We’re still shocked to this day that we were that lucky.”