April 2026 – Utah Rare Disease Advisory Council

DENALI THERAPEUTICS ANNOUNCES U.S. FDA APPROVAL OF AVLAYAH™ (TIVIDENOFUSP ALFA-EKNM) FOR TREATMENT OF HUNTER SYNDROME (MPS II)

Posted on April 28, 2026 by admin

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

Posted on April 28, 2026 by admin

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

Posted on April 25, 2026 by admin

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

Posted on April 15, 2026 by admin

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

Posted on April 25, 2026 by admin

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 […]