Congenital, Hereditary, and Neonatal Diseases and Abnormalities: News and Research on Mucopolysaccharidosis II

MeSH tree: C16.320.400.525.750

Enzyme replacement therapy offers hope for ultra-rare Hunter syndrome

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic decline.

Jan 5, 2026

0

57

First ever gene therapy trial for children with Hunter syndrome opens

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.

Oct 30, 2023

0

43