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.

Comparison of the disease course in patients with the severe and mild forms of Hunter's syndrome indicates that the severely affected boys show a higher incidence of behavioural disorder, diarrhoea, ...

The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder. Five ...

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

A case of Hunter syndrome, 6½-year-old boy presented with persistent thrombocytopenia ... and bone marrow done then showed-foamy macrophages suggestive of storage disorder. IQ done earlier was 70. The ...

The significant partnership agreement is for the clinical development of an investigational lentiviral gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome, a rare and deadly ...