High Stakes for Hope

The night

Same room that already proved it can move the science.

Last year’s Starlight Soirée brought leaders and families to Via Tivoli and raised about $50,000 toward the next stage of gene-therapy work. High Stakes for Hope is the next Rare Disease Day night in that house: sunset over the valley, a black-tie Casino Royale, and a clear ask.

Last year’s room funded the first phase of this work. The full next-stage study needs about $2.5 million over three years. This night is one piece of that runway — not a one-year budget that finishes the job.

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What the money funds

Not a slogan. A therapy that has already worked in the liver.

Life For Liam And Friends Foundation funds the Schultz Laboratory at the University of Iowa Stead Family Children’s Hospital. The mRNA work has already restored function in the liver. The next stage is getting that same idea to the brain — a three-year study that needs about $2.5 million. This night does not cover that by itself.

NPC is often called childhood Alzheimer’s. Solving delivery of mRNA to the brain for Liam also opens a path for Alzheimer’s, Parkinson’s, and thousands of other rare genetic diseases.