First in-human, intracisternal dosing of RGX-111, an investigational AAV gene therapy, for a 21-month-old child with mucopolysaccharidosis type I (MPS I): 3.5 year follow-up
Raymond Wang(Children's Hospital of Orange County), Laura Rosa Pisani(Regenxbio (United States)), Richard Chang(Maryland Oncology Hematology), Mery Taylor, Paulo Falabella, Nina Movsesyan, Tammam Beydoun, Dawn Phillips, Jenna Burke(Regenxbio (United States)), Yoonjin Cho, Michelle Gilmor, Shih‐hsin Kan
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