RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)

key information

study id #: NCT03566043

condition: Mucopolysaccharidosis Type II (MPS II)

status: Recruiting


RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a safety and dose ranging study to determine whether RGX-121 is safe and tolerated by patients with MPS II.

intervention: RGX-121


last updated: February 26, 2022