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 ...

Before Ryann was born, a prenatal screening revealed he had a genetic disorder known as Hunter Syndrome or Mucopolysaccharidosis Type II. It is a rare genetic disorder that Queen's little brother ...

SHP609 was supposed to correct the deficiency in the iduronate-2-sulfatase enzyme in the CNS and prevent the cognitive decline seen in some Hunter Syndrome cases. Sadly, Shire said the phase 2/3 ...

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 ...