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

In 2021, the FDA granted Fast Track designation to tividenofusp alfa for the treatment of patients with Hunter syndrome. In 2022, the European Medicines Agency granted Priority Medicines ...

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 ...

Enzyme replacement therapy is often used as treatment for the disorders ... to complete submission of its application for the Hunter syndrome therapy, called RGX-121, for FDA approval this ...

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.

That's because it recently received FDA Breakthrough Therapy Designation [BTD] for tividenofusp alfa [DNL310] for the treatment of patients with Hunter Syndrome [also known as MPS II]. Even better ...

One of the treatments, for a progressive disorder known as Hunter syndrome, could secure approval in the U.S. as soon as late this year. Should that happen, Regenxbio will retain rights to any ...

Dominic travels to Chicago every month to receive his treatments. Along the way, his family has connected with other families affected by Hunter syndrome, sharing their stories and offering ...