RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)
study id #: NCT04571970
condition: Mucopolysaccharidosis Type II (MPS II)
status: Recruiting
purpose:RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine whether RGX-121 is safe, well tolerated, and potentially effective in children five years of age and over who have severe MPS II.
intervention: RGX-121
results: https://clinicaltrials.gov/ct2/show/results/NCT04571970
last updated: February 26, 2022
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