If I recall correctly, they started this 2 year ago, when she was turning 5. She was loosing skills, on her way to dying. The US government hasn’t funded cure research for Sanfilipino because of how few people (all children) are effected (literally less than 10 a year) and government funding money has to go to the places it will help the most people. This family refused that fate, and along with others organized fundraising that brought in the $2 million necessary to start production on a gene therapy drug for human trials. The only existing possibly promising treatment to save their child’s life, but untested and unproven and even non-existent, it was only a theory. It took almost twice as long as the 9 months they expected to get to trials, and they had to watch their daughter slipping away. Children with this disease don’t live much past 6 years old. She could have been lost, and sadly a couple children that started this already have been lost. But the therapy trial finally came through, and Eliza was one of two children treated, and now she is showing positive progress! Slowly relearning how to speak and other skills!
With last year’s fundraiser, a handful more children were to be treated. This IS literally saving children’s lives, one at a time. Together we can save one more. And while that may only be one child, every one child is someone’s child, is our child.