After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...

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

Long-term data demonstrate that robust reductions and normalization in key biomarkers from baseline were maintained over time with continued ...

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 ...

Shire’s attempts to develop a drug for cognitive impairment from the rare inherited disease Hunter Syndrome has failed to produce results. The company’s Elaprase (idursulfase) is already ...

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 ...