CAMPSIITE® Phase I/II/III: Interim clinical update of clemidsogene lanparvovec (RGX-121), an investigational gene therapy for treatment of neuronopathic mucopolysaccharidosis type II (MPS II)
Roberto Giugliani, M.D., Ph.D., Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, Brazil