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 ...

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 ...

About DNL310 and Hunter Syndrome (MPS II) Hunter syndrome (MPS II) is a rare neurodegenerative lysosomal storage disease caused by mutations in the gene that encodes for the enzyme iduronate-2 ...