University of Manchester5mon
Groundbreaking gene therapy trial for 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. Five ...
HealthPrep on MSN11mon
How To Treat Hunter Syndrome
Hunter syndrome is an exceedingly rare genetic disorder caused by a malfunctioning or missing enzyme. Patients with Hunter ...
manilatimes1mon
Denali Therapeutics Announces Primary Analysis and Long-Term Follow-Up of Phase 1/2 Study in Hunter Syndrome (MPS II) with Tividenofusp Alfa
Denali also supported additional research relating to Hunter syndrome (MPS II) at the WORLDSymposium™ conference. Research presented by Barbara Burton, M.D., Professor of Pediatrics, Genetics, ...