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

One of the treatments, for a progressive disorder known as Hunter syndrome, could secure approval in the U.S. as soon as late ...

Hunter syndrome results from the absence of an enzyme needed to break down cellular waste. Without it, waste builds up in the body, causing progressive damage to various systems.

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 ...

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 ...

Denali obtains Breakthrough Therapy Designation for its experimental candidate, tividenofusp alfa (DNL310), for the treatment of pateints with Hunter syndrome.

Haliza Md Kasim’s eyes widened. Despite having gone through this before with her first son, Yusof, and despite the doctors ...

Regenxbio (RGNX) and Nippon Shinyaku announced a strategic partnership for the development and commercialization of RGX-121 for the treatment ...

REGENXBIO Inc. (RGNX) and Nippon Shinyaku announced a strategic partnership for the development and commercialization of RGX-121 for ...