28 May 2023 - Imagine having two daughters who both have a rare disease and watching them struggle — to be understood, to remember things, to learn, to be comfortable in social settings, to follow one-step directions, to be able to bond with friends and family.

Now, imagine a clinical trial showed promise for your older daughter. You notice a remarkable difference in her daily living, and suddenly, your home is more peaceful, and she is starting to thrive. The treatment is potentially life-changing. You now have hope for your older daughter and her younger sister.

Read The Hill article