RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

Hunter Syndrome is a rare genetic condition that can cause damage to the body and the brain. It often causes children to pass away in their teens, but a UNC Health ...

In a complete response letter published by the FDA on Monday, the agency said a resubmission for REGENXBIO’s Hunter syndrome gene therapy should provide evidence of normalized or improved biomarker ...

A new genetic disorder, Hunter syndrome, has been added to Utah’s Newborn Screening Program, bringing the total to 45 conditions that can be identified shortly after a child is born — and before ...

All McKay wants this holiday season is for new therapies for those with Hunter syndrome to be approved. "We really want the FDA to understand the urgency that putting a delay 'just three months,' for ...

SALT LAKE CITY — New parents may cringe thinking about the heel pricks routinely done after a baby is born and then again two weeks later, but the required screening saves lives. "I have had cases ...

A new Prescription Drug User Fee Act target date of February 8, 2026 has been set for the application. The Food and Drug Administration (FDA) has extended the review for the Biologics License ...

(RTTNews) - Denali Therapeutics Inc. (DNLI) on Monday announced that the U.S. Food and Drug Administration has extended its review timeline of the company's investigational therapeutic Tividenofusp ...

Ollie Chu was three years old when he received an infusion that would change his life. Born with a rare inherited condition called Hunter syndrome, Ollie’s body couldn’t produce an enzyme that breaks ...