Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio— have products in the pipeline that could ...

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 ...

Hunter syndrome can be diagnosed using the physical symptoms of children at the age of 18 months and 4 years. Treatments can help manage condition, no known cure Diagnosed by medical professional ...

With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now found a commercialization partner for both the U.S. and Asian markets.

RegenxBio said Tuesday that Kyoto, Japan’s Nippon Shinyaku Co. Ltd. will develop and commercialize its one-time gene therapies for Hunter syndrome and Hurler syndrome, both affecting children.