Skip to main content

One Minute Summary of May 2024 Patient Forum


My daughter’s name is Amara. She just turned eight years old, and she suffers from FOXG1 Syndrome, which is an ultra-rare neurodevelopmental condition.

Our only child, Ryan, was diagnosed with an ultra-rare condition called Mucopolysaccharidosis Type 1.

I’m actually a second career nurse.

So when I began, you know, the FOXG1 Research Foundation with a group of parents, we really act like a mini biotech.

We were told at his diagnosis that, Ryan’s lifespan would be shortened to within a decade. He was three and a half at the time.

I am in the Doctorate in Nursing Practice program at the University of California, San Francisco, and I’m really looking forward to, becoming the first person in my family on either side to achieve a doctorate degree.

We developed our own mouse models and cell lines. We started experimenting with different gene therapies and antisense therapies. We built a small molecule screening platform.

The pathway towards accelerated approval is through a surrogate biomarker.

To support our novice nurses as their mentees, having cultural sensitivity and humility when they are providing equitable care to a diverse patient population.