"We were not in trials. We started exon 51 2 months back. We do see a lot of difference since we started on exon 51. At 4 years old he had trouble climbing even a toy slide. But now he is climbing the slide in park in reverse. No falling anymore and…"
"My son has had his port for a little over a year (he was in the trial). It's been relatively smooth. It freaked him out at first (he'd just gotten used to arm pokes) but infusions are no big deal now. He does…"