P. Harmatz, Can Ficicioglu, Roberto Giugliani, Deepa Rajan, Joseph Hagood, M. Fiscella, Lin Yang, Michelle Gilmor, Yoonjin Cho, Caroline Mulatya, Dawn Phillips, Nidal Boulos, Paulo Falabella, Laura Pisani
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CAMPSIITE™ phase I/II/III: An interim clinical study update of RGX-121, an investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II)