With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now ...

The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder. Five ...

People genetically susceptible to Huntington's disease often see their movement, mood, and cognition decline slowly over time.

In our research, we screened nearly 10,000 DNA samples from across the world, dating as far back as when humans were hunter-gatherers. The six individuals we identified with Down syndrome were all ...

Fifteen-year-old Dominic Henriquez from Prosper, Texas, is bringing hope to boys like him who live with Hunter syndrome, a rare genetic disorder that primarily affects males. Dominic’s journey ...

In a deal potentially worth $810 million for Regenxbio Inc., Nippon Shinyaku Co. Ltd. is partnering on the U.S. and Asian development and commercialization of iduronate-2-sulfatase enzyme RGX-121 for ...

The University of Manchester has announced today a groundbreaking gene therapy partnership to ease the lifelong suffering of people with Hunter syndrome. The University has agreed to a worldwide ...