Marian is the youngest patient in the world that we know of to be receiving this treatment via IT (spinal fluid). Another title holder is our friend Parker who is only six months old and just started it but through an IV to target his liver. Spain allows compassionate...

When I was little my best friend Ashley and I used the same after school babysitter. In second and third grade we would wait in the elementary school circle driveway waiting for her to come get us. Sometimes she would be late. I would turn to Ashley and nervously ask,...