First-in-human intracisternal dosing of RGX-111 in severe MPS I is well tolerated and generates sustained neurodevelopment without HSCT
Raymond Wang(Children's Hospital of Orange County), Laura Rosa Pisani(Regenxbio (United States)), Nina Movsesyan, Tammam Beydoun, Jenna Burke(Regenxbio (United States)), Mery Taylor(Children's Hospital of Orange County), Michelle Gilmor(Regenxbio (United States)), Richard C. Chang(Children's Hospital of Orange County), Yoonjin Cho(Regenxbio (United States)), Shih‐hsin Kan, Dawn Phillips(Regenxbio (United States)), Paulo Falabella
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