DENALI THERAPEUTICS ANNOUNCES U.S. FDA APPROVAL OF AVLAYAH™ (TIVIDENOFUSP ALFA-EKNM) FOR TREATMENT OF HUNTER SYNDROME (MPS II)
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 […]
Utah Rare Disease Advisory Council Gets New Leadership
Gina Szajnuk to Replace Dr. Lorenzo Botta as RDAC Chair Utah’s Rare Disease Advisory Council (RDAC), administered by BioUtah, in conjunction with the Utah Department of Health and Human Services, met on July 15 to conduct business and transition to a new leadership team. Stepping down as RDAC Chair after four years of exemplary service […]
Stakeholder Feature: Dr. Botto
Each quarter, we highlight a council member, an advocate or policy maker, an investigator, or clinical care provider. This quarter we are featuring one of the Utah RDAC members, Dr. Lorenzo Botto. “My career has taken me across several countries and disciplines, shaped by the mentors, patients, and families I have learned from along the […]
Tayler’s Battle with Pulmonary Hypertension
We hope to strengthen connections in the community through personal stories that describe barriers or challenges that a rare disease patient or family has encountered. We will evaluate how the barrier was cleared. If the challenge has not been resolved, we will explore what is needed to solve that problem. This quarter, we will describe […]
