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RGX-121 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type II (MPS II): Interim Analysis of Data from the First in Human Study

D. R. Blair,P. Harmatz,C. Ficicioglu,R. Giugliani,D. S. Rajan,J. Hagood,M. Fiscella,L. Yang,M. Gilmor,Y. Cho, C. Mulatya,D. Phillips,P. Falabella, L. Pisani

JOURNAL OF INVESTIGATIVE MEDICINE（2024）

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Key words
Rare Diseases,Neurometabolic Disorders
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