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 ...

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

Japan’s Nippon Shinyaku is continuing to scoop up the international rights to some intriguing drugs, this time penning a $686 ...

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

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.

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 ...