I don’t mean to sound aggressive in my responses but even with intervention, there is no cure and it’s fatal. Intervention may slightly increase life expectancy, but even then, imagine an individual who is stuck in the body of a teenager but their brain is so deteriorated they can’t walk, talk, sit up, communicate, eat, or drink. That’s what happens at the end when you’re looking at this disease, intervention or not. I pray they find a cure.

How is it a relief for a child to be diagnosed with a fatal disease that, on average, won’t allow them to live past adolescence? And even then, the quality of life is an absolute tragedy. The brain starts deteriorating before it’s even had a chance to develop causing the entire body to slowly shut down over time because the brain stops sending signals for basic functions like digesting food and the ability to move. I work with parents of an individual with Sanfilippo, who was first misdiagnosed as autistic, and also received the stem cell transplant a few years ago and it sadly had no positive effect. This child just had a birthday and according to their doctors it will almost definitely be their last. When you see the horror of the disease firsthand you realize the diagnosis, early or not, translates directly to early death. No child or family deserves that.