About:serfwerks

Website: https://utahrdac.org

Posts by serfwerks :

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

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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 […]

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Utah Rare Disease Advisory Council Gets New Leadership

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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 […]

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Stakeholder Feature: Dr. Botto

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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 […]

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Tayler’s Battle with Pulmonary Hypertension

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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 […]

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