By Dr. Sanchari Sinha Dutta, Ph.D. Study uncovers how gradual CAG repeat expansion in neurons drives the onset of ...

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

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

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.

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

The partnership between REGENXBIO and Nippon Shinyaku is more than just a standard collaboration—it’s a bold, forward-looking venture aimed at bringing revolutionary gene therapies to market for rare, ...

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

In 2021, the FDA granted Fast Track designation to tividenofusp alfa for the treatment of patients with Hunter syndrome ... mutations confirmed by genetic testing. BIIB122 is also being evaluated ...

This designation is in addition to Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation, all previously granted by the FDA for tividenofusp alfa in Hunter syndrome.