Raymond Y. Wang, Nina Movsesyan, Shih-hsin Kan, T. Beydoun, M. Taylor, Richard C. Chang, Dawn Phillips, Jenna Burke, Michelle Gilmor, Yoonjin Cho, Paulo Falabella, Laura Pisani
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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