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 ...

Attorneys for Johns Hopkins All Children's Hospital filed an appeal in the "Take Care of Maya" case, arguing they didn't receive a fair trial.

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 ...

has awarded Breakthrough Therapy Designation to its investigational drug tividenofusp alfa for the treatment of Hunter syndrome, also known as MPS II. This recognition follows previously granted ...

A new poll from The Associated Press-NORC Center for Public Affairs Research shows about half of U.S. adults believe it is a ...

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 ...