We received Emmy’s diagnosis shortly after her first birthday and it came as a complete shock to have someone tell us that our seemingly normal, happy toddler has a serious genetic disease with no cure. But thanks to the love, support, and generosity of family, friends, and people like you, we continue to have hope that life-improving advances will be made and hopefully a cure will be found in the near future for our amazing daughter and the hundreds of other children affected by this miserable disease.