Presentation & Publications

RGX-121 Gene Therapy For The Treatment Of Neuronopathic Mucopolysaccharidosis Type II (MPS II): Interim Analysis Of Data From The First In Human Study (Abstract #LB-21)

Can Ficicioglu, M.D., Ph.D., Professor of Pediatrics at Perelman School of Medicine at the University of Pennsylvania, Director of the Newborn Metabolic Screening Program and the Lysosomal Storage Diseases Program, Clinical Director of the Metabolic Disease Program Children's Hospital of Philadelphia