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

with Mucopolysaccharidosis Type II (MPS II), which is also known as Hunter syndrome. The pivotal phase of the study met its primary endpoint of patients achieving a reduction in cerebrospinal ...

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 ...

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).

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

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 ...

“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 ...

Hunter syndrome results from the absence of an enzyme ... treatment options and exploring clinical trials. While there is no cure, Dominic was enrolled in an experimental trial that delivers ...

Enzyme replacement therapy is often used as treatment for the disorders, which don’t have a cure. The transaction ... of its application for the Hunter syndrome therapy, called RGX-121, for ...

We are grateful to the FDA for recognizing the potential of tividenofusp alfa as a meaningful treatment option for individuals with Hunter syndrome. We look forward to continued collaboration with ...