For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

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

Mucopolysaccharidosis Type II (MPS II) also known as Hunter Syndrome is when the body lacks the enzyme to break down sugar ...

Access to genetic testing brings with it several advantages – one being early diagnosis for young patients with potentially life-threatening multi-system conditions. Retinal-renal syndrome ...

In addition to opening a new window into how human genetic mutations can operate, the researchers hope that their insights lead to new strategies that improve or ultimately save the lives of people ...

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 ...

The study identifies 106 genetic variants linked to occupational status, highlighting the interplay between genetics, family environment, and childhood aspirations.

One month after the movie flopped in theaters, Sony’s latest Marvel release, Kraven The Hunter, is now available to stream on digital. Keep reading to find out how you can watch Kraven the ...

Nip­pon Shinyaku has paid $110 mil­lion up­front for the rights to sell two of Re­genxbio’s gene ther­a­pies for mu­copolysac­cha­ri­do­sis in the US and Asia. … ...

Japan’s Nippon Shinyaku Co. Ltd. will develop and commercialize its one-time gene therapies for Hunter syndrome and Hurler syndrome, both affecting children. Both are rare genetic disorders ...

Scientists have found 697 genetic variants – or small changes within a gene – linked to depression, of which almost 300 were previously unknown. A Queensland scientist described the project as ...

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.