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

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 ...

Shire’s attempts to develop a drug for cognitive impairment from the rare inherited disease Hunter Syndrome has failed to produce results. The company’s Elaprase (idursulfase) is already ...

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 ...

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.

We are grateful to the FDA for recognizing the potential of tividenofusp alfa as a meaningful treatment option for individuals with Hunter syndrome. We look forward to continued collaboration with ...