RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)
study id #: NCT03566043
condition: Mucopolysaccharidosis Type II (MPS II)
status: Recruiting
purpose: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
results: https://clinicaltrials.gov/ct2/show/results/NCT03566043
last updated: February 26, 2022
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