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 ...

Hunter syndrome — also known as mucopolysaccharidosis type II, or MPS II — worldwide affects more than 2,000 people, mainly males. The inherited disorder leads to a buildup of sugar molecules ...

"This regulatory milestone brings us closer to our goal of delivering a new treatment option to the Hunter syndrome community as we continue to listen, learn, and seek their advice in bringing ...

“Mice with Hunter syndrome treated with the HSC gene therapy showed dramatic improvement in their condition, including normalization of working memory problems, and skeletal features such as the ...

for accelerated approval of tividenofusp alfa for the treatment of Hunter syndrome (MPS II). The same has also been received by the Center for Drug Evaluation and Research (CDER) of the FDA.

Tividenofusp alfa is designed to address both cognitive and physical symptoms of Hunter syndrome, a rare genetic disorder primarily affecting males worldwide. The current standard of care does not ...