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- [30] First in Human RESKUE Phase 1/2 Clinical Trial of Intravenous FBX-101 (AAVrh10. hGALC) Administered after Immune and Myeloablation for Unrelated Umbilical Cord Blood Transplantation Prevented Immune Responses, Increased GALC Activity, Restored Normal Brain Development, and Normalized Motor Function in Patients with Infantile Krabbe Disease MOLECULAR THERAPY, 2023, 31 (04) : 193 - 194