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

scores in children with Hunter syndrome after 12 months of treatment. The key secondary endpoint evaluated the difference between the SHP609-treated and control groups as measured by the change ...

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 ...

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 ...

Despite having gone through this before with her first son, Yusof, and despite the doctors explaining that this was a gene ...

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 ...

Denali’s wholly owned program, DNL310 or tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy in development for MPS II (Hunter syndrome).

Hunter’s syndrome affects only about 1 in 162,000 ... life expectancy has been raised to 19 years old, however– lots of children pass with this disease around age 13. For McKay, the community ...

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.