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.

for the treatment of patients (up to five years old) with Mucopolysaccharidosis Type II (MPS II), which is also known as Hunter syndrome. The pivotal phase of the study met its primary endpoint of ...

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

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