Denali Therapeutics Inc. DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, tividenofusp alfa (DNL310), for the treatment of individuals with Hunter ...

With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now ...

Now, they have a treatment that arrests or halts the disease process,” Muenzer said. Muenzer is recognized as one of the world’s leading experts in Hunter syndrome research and the medical ...

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

Nippon Shinyaku pays $110M upfront to Regenxbio for US/Asia rights to RGX-121 and RGX-111 gene therapies for ...

scores in children with Hunter syndrome after 12 months of treatment. The key secondary endpoint evaluated the difference between the SHP609-treated and control groups as measured by the change ...

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 this year. Should that happen, Regenxbio will retain rights to any ...