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 ...

Hunter syndrome results from the absence of an enzyme needed to break down cellular waste. Without it, waste builds up in the body, causing progressive damage to various systems.

This is a retrospective study of the history, roentgenology and autopsy pathology when appropriate of all infants with severe respiratory-distress syndrome who required twenty-four hours or more ...

With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now ...

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 ...

Hunter syndrome, which affects an estimated one in 100,000 males worldwide, causes devastating complications throughout the body and brain, including severe cardiac and respiratory dysfunction, ...