This spring, our family learned some very devastating news - our Lucy (my niece) was diagnosed with a rare, devastating genetic neurologic disease. 

This video shares the story:

We have been told she would likely never walk or talk and would probably develop severe untreatable seizures in early childhood. Her parents (my brother and sister in law) looked at their happy, smiling, curious, babbling, cruising baby and defiantly decided that didn’t have to be her story.

Geri and Zach connected with researchers all over the world, and they are doing everything they possibly can to drive the development of a gene therapy treatment that would give Lucy and other kids suffering from PGAP3 back a normal copy of the PGAP3 gene. It's pretty amazing – in the 5 months since her diagnosis, they’ve formed a 501c3 non-profit, Moonshots for Unicorns, and enlisted the gene therapy team at Nationwide Children’s Hospital led by Dr. Kat Meyer who are rapidly making progress toward a cure. The science is moving fast – already, Lucy’s skin cells have been turned into neurons and astrocytes, there are PGAP3 mice running around in Maine ready for therapies to test, and the gene therapy DNA is being placed into a harmless virus for delivery. The next phase of the project – manufacturing the gene therapy virus and testing it for safety and efficacy – will start in 2023 and they need $1.3M more to make it happen.

This campaign is to raise the money to fund the gene therapy project. The total cost is about $2.5M, but if anyone can do it, these parents can – especially if we can help them.  The goal of  1.3 by 2023 is to fund the next phase of the research, after which point, pharma/biotech can come in and run the rest of the way through expensive clinical trials and commercialization.

Asking for help is hard but not when its for such an important cause. Please help make Lucy's cure a reality for our family and many other families with children suffering from PGAP3 disorder. Any donation amount helps. Thank you to the Moon and Back!