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Stem Cell Transplant
Gene Therapy vs Stem Cell Treatment for Hurler Syndrome
Recruiting2 awardsPhase 3
Oakland, California
This trial compares a new gene therapy called OTL-203 with traditional stem cell transplants in patients with a severe genetic disorder called MPS-IH. The goal is to see if gene therapy can better correct the genetic defect causing the disease compared to replacing damaged cells with healthy ones from a donor.
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