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)
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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