I am excited to hear this news too! We go to St. Louis Children's Hospital (Washington University) and my daughter's doctor said it is looking really good in the trials and that it would essentially be a "cure" for those with the nonsense mutations. I didn't know about the pediatric trials starting soon.... I'll have to try and get Kate in that. My daughter is only two, so this is just thrilling stuff!!!
Zoe, mom to Kate wCF
Zoe, mom to Kate wCF