with Mucopolysaccharidosis Type II (MPS II), which is also known as Hunter syndrome. The pivotal phase of the study met its primary endpoint of patients achieving a reduction in cerebrospinal ...

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

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

REGENXBIO's partnership with Nippon Shinyaku provides $110M upfront, ensuring a cash runway for gene therapy programs in MPS ...

In a deal potentially worth $810 million for Regenxbio Inc., Nippon Shinyaku Co. Ltd. is partnering on the U.S. and Asian development and commercialization of iduronate-2-sulfatase enzyme RGX-121 for ...