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RGX-121-5101 is the long-term follow-up study to the RGX-121-101 first in human study where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfa...
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Lead Sponsor
REGENXBIO Inc.
NCT02254863 · Adrenoleukodystrophy, Batten Disease, and more
NCT03153319 · Mucopolysaccharidosis I, Mucopolysaccharidosis II, and more
NCT04348136 · Mucopolysaccharidosis II
NCT05422482 · Mucopolysaccharidosis II, Hunter Syndrome
NCT06333041 · Sanfilippo Syndrome, Mucopolysaccharidosis III
Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders
Pittsburgh, Pennsylvania
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This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
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