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 ...

According to the Theodore Roosevelt Conservation Partnership (TRCP), a large national coalition of hunting and fishing groups, always-fatal Chronic Wasting Disease (CWD) is the biggest threat to the ...

PAUL HUNTER was an iconic snooker player when he was alive and his legacy has continued long after his death aged just 27. Every year at the Masters tournament the sportsman is remembered ...

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.

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 ...