Stakeholder Feature: Primary Children’s Gene Kids & Center for Personalized Medicine
Primary Children’s Gene Kids is a $9 million program to provide a genomic diagnosis to every child with a possible genetic condition. Our goal is to integrate diagnosis, discovery, and treatment to reach children and families who may benefit, regardless of their location or insurance, by ensuring access to whole genome sequencing (WGS). Gene Kids […]
Stakeholders, Partners, and Communications Workgroup Report
The Stakeholders, Partners, and Communications Workgroup has been focused on expanding awareness of the Utah Rare Disease Advisory Council (RDAC), strengthening relationships with key stakeholders, and increasing engagement with both policymakers and the rare disease community. One of our primary goals has been to help more individuals and organizations understand who the Utah RDAC […]
RDAC Member Feature: Marian Furst
With a brand new PhD in geochemistry, I joined an oilfield service company, spending four years on special projects in the company research lab in Connecticut and two years as a geochemist advisor to the operations part of the company in Houston and Dallas. When oilfield drilling activity in the US plummeted, I was laid […]
RDAC at Canyon Rim Cares
On July 18, 2026, members of the Utah Rare Disease Advisory Council (RDAC) were honored to participate in Canyon Rim Cares, an annual day of service that brought together nonprofit organizations, volunteers, community leaders, and residents who share a commitment to strengthening our communities through service and connection. Throughout the event, RDAC members had the […]
Rare Voices: Sarah & Andrew Jeffs
We gave our youngest son the name of Andrew because it means “strong and manly.” When I was pregnant with him, a routine ultrasound showed that he would be born with clubbed feet. We knew then that he would need all of the strength he could get. Shortly after he was born, we took him […]
Denali Therapeutics Announces FDA Approval of Treatment for Hunter Syndrome
First new FDA-approved treatment option in nearly 20 years for families living with this rare lysosomal storage disease First FDA-approved medicine in emerging new class of biotherapeutics that leverage transferrin receptor to cross blood-brain barrier Denali’s first medicine enabled by its TransportVehicle™ platform designed to deliver biotherapeutics to whole body, including brain Rare Pediatric Disease […]
RDAC, Governor Cox & The Utah Jazz Honor Rare Disease Day 2026
Thank you to the Utah Jazz and Governor Cox for honoring Rare Disease Day and the work of the Rare and Undiagnosed Network (RUN) and the Utah Rare Disease Advisory Council! It was a very special evening for all of us. It was wonderful to see our rare families. There were a lot of hugs […]
RDAC Policy and Legislative Workgroup Report, Utah General Session 2026
More than 1,000 bills and resolutions were introduced during the 45-day Utah legislative session from January 20 to March 6. The RDAC Legislative Committee screened all bills and resolutions for relevance to the rare and undiagnosed disease community and prioritized its efforts based on the RDAC Policy Guide and subject matter it deemed urgent in […]
Stakeholder Feature: Recursion
Recursion is a Salt Lake City–based company using AI and scientific innovation to discover and develop new medicines for people living with diseases that often have few or no treatment options, including rare diseases. Around the world, an estimated 300 million people are affected by a rare disease, yet only about 5% of rare diseases […]
Courageous Hope: Action Before Answers
By: Stacy Allen Most people think of hope as something passive, a feeling you hold onto while waiting for things to improve. But real hope, the kind that changes outcomes, looks very different. It’s active. It’s uncomfortable. And often, it shows up before there are any answers at all. That’s what I’ve come to understand […]