New drug offers hope for children with Hunter syndrome
The NC TraCS Clinical & Translational Research Center (CTRC) provided space and highly skilled nursing staff for this clinical trial.
Two North Carolina families share their journey with the rare genetic disorder and the potential impact of the breakthrough treatment in a national television news story.
Atlas and Cole are two North Carolina boys living with Hunter syndrome, a rare genetic disorder.
In this national TV story with Investigative TV+, their families share the challenges the boys face as well as the hope they have for the future thanks to the first major advance in the care of Hunter syndrome in 20 years.
The new drug called Avlayah™, is developed by Denali Therapeutics. The initial results of a clinical trial on the drug were published in the New England Journal of Medicine in January 2026.
See also: UNC Research contributes to FDA approval of new drug for rare genetic disorder
Media contact:
Originally posted at news.unchealthcare.org