He was born with a deadly mutation. Delivered five weeks early to start gene therapy at SickKids, his progress is offering new hope for infants diagnosed with the severe form of spinal muscular atrophy (SMA Type 1). This story is about a baby boy named David, the youngest patient in Canada to receive gene therapy for SMA Type 1, and the dedicated medical team at the Hospital for Sick Children (SickKids) in Toronto who are changing the course of this debilitating disease.
David’s Journey: A Beacon of Hope
Dr. Hernan Gonorazky, director of the Neuromuscular Program at SickKids, is visibly pleased as he examines nine-month-old David, observing his normal development. Just a few years ago, Dr. Gonorazky wouldn’t have been able to deliver such good news. The most severe form of spinal muscular atrophy, SMA Type 1, typically results in babies not surviving past the age of two. They often require breathing and feeding support within their first few months of life.
However, David’s case is different

