hunter-syndrome-mps-ii Clinical Trials
A listing of hunter-syndrome-mps-ii medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Found 1 clinical trials
D
Debra L Day-Salvatore, MD
RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety, efficacy, and pharmacodynamic dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II (Hunter Syndrome)
