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

for the treatment of patients (up to five years old) with Mucopolysaccharidosis Type II (MPS II), which is also known as Hunter syndrome. The pivotal phase of the study met its primary endpoint of ...

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 ...

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

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 ...

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).

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.

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 ...