RGX-111 Gene Therapy in Patients With MPS I

key information

study id #: NCT03580083

condition: Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome

status: Recruiting


RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system. This is a safety and dose ranging study to determine whether RGX-111 is safe and tolerated by patients with MPS I.

intervention: RGX-111


last updated: February 26, 2022