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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 use and my high school spanish is ever waning, so it's possible there's someone in Spain as young as Marian. However, within the United States and most of the world it's definitely Marian Linda McGlocklin. It's

Marian has ultra high frequency hearing loss. We sat in the little room together for the hearing test. Not to brag but I could hear all the sounds, but Marian couldn't. She sat in my lap as they played sounds from speakers on either side of her. A little on the left, a little on the right, and a toy would light up if she looked the correct direction. When the higher sounds played she didn't respond, they went a little louder, and no response still, they went a little louder a

I sometimes would sit at night looking at charts for NPC. There's a white line for pre-neurological symptoms, a grey line for neurological symptoms and sometimes a blue line for attempted interventions. Sometimes these lines are long, usually when there's no symptoms until kids are seven are eight. Marian is still only one years old, she doesn't have typical neurological symptoms but does have developmental delays - which means it's been in her brain somewhere already. Warnin