Update on RGX-121 Treatment for MPS II

The National MPS Society is pleased to share an update on RGX-121, an investigational gene therapy for MPS II (also known as Hunter Syndrome), from REGENXBIO!

“A potential one-time treatment that can allow these boys to exceed the natural history of this disease in their neurocognitive development, as well as the ability to remain off enzyme replacement therapy for multiple years represents a meaningful option for patients and their families,” said Roberto Giugliani, M.D., Ph.D., Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, Brazil. “I continue to be very encouraged by the data supporting RGX-121 and look forward to seeing this program advance towards potential approval for this community.”

You can read a statement from REGENXBIO below, or you can click here to read more about the therapy and its trial results on their website.