Chromosomes showed del(17)(q23.1-->q24.2); the more severe phenotype may be explained by the deletion. The deletion also suggests the possibility that the gene for Hunter-McAlpine syndrome might ...

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

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

Mutations in the X chromosome often cause intellectual disability, and women born with just one X chromosome (a diagnosis ...

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.

Most people carry two "versions" of the first 22 chromosomes ... as far back as when humans were hunter-gatherers. The six individuals we identified with Down syndrome were all from Europe ...

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).

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.