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 ...

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 ...

Jeffrey Hung, an analyst from Morgan Stanley, maintained the Buy rating on Denali Therapeutics (DNLI – Research Report). The associated price ...

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

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 ...