Enzyme replacement therapy for CLN2 disease: MRI volumetry shows significantly slower volume loss compared to a natural history cohort

From the Department of Radiology, Great Ormond Street Hospital, London, UK (PG, AB, KM, SS, FD, UL), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London, London, UK (PG), Department of Paediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (AS), Department of Diagnostic and Interventional Neuroradiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (JF), Robert Steiner MR Facility, Medical Research Council Laboratory of Medical Sciences, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS) and the Mansfield Centre for Innovation, Imaging Sciences, Institute of Clinical Sciences, Imperial College London, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS).

From the Department of Radiology, Great Ormond Street Hospital, London, UK (PG, AB, KM, SS, FD, UL), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London, London, UK (PG), Department of Paediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (AS), Department of Diagnostic and Interventional Neuroradiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (JF), Robert Steiner MR Facility, Medical Research Council Laboratory of Medical Sciences, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS) and the Mansfield Centre for Innovation, Imaging Sciences, Institute of Clinical Sciences, Imperial College London, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS).

From the Department of Radiology, Great Ormond Street Hospital, London, UK (PG, AB, KM, SS, FD, UL), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London, London, UK (PG), Department of Paediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (AS), Department of Diagnostic and Interventional Neuroradiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (JF), Robert Steiner MR Facility, Medical Research Council Laboratory of Medical Sciences, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS) and the Mansfield Centre for Innovation, Imaging Sciences, Institute of Clinical Sciences, Imperial College London, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS).

From the Department of Radiology, Great Ormond Street Hospital, London, UK (PG, AB, KM, SS, FD, UL), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London, London, UK (PG), Department of Paediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (AS), Department of Diagnostic and Interventional Neuroradiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (JF), Robert Steiner MR Facility, Medical Research Council Laboratory of Medical Sciences, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS) and the Mansfield Centre for Innovation, Imaging Sciences, Institute of Clinical Sciences, Imperial College London, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS).

From the Department of Radiology, Great Ormond Street Hospital, London, UK (PG, AB, KM, SS, FD, UL), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London, London, UK (PG), Department of Paediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (AS), Department of Diagnostic and Interventional Neuroradiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (JF), Robert Steiner MR Facility, Medical Research Council Laboratory of Medical Sciences, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS) and the Mansfield Centre for Innovation, Imaging Sciences, Institute of Clinical Sciences, Imperial College London, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS).

From the Department of Radiology, Great Ormond Street Hospital, London, UK (PG, AB, KM, SS, FD, UL), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London, London, UK (PG), Department of Paediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (AS), Department of Diagnostic and Interventional Neuroradiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (JF), Robert Steiner MR Facility, Medical Research Council Laboratory of Medical Sciences, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS) and the Mansfield Centre for Innovation, Imaging Sciences, Institute of Clinical Sciences, Imperial College London, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS).

From the Department of Radiology, Great Ormond Street Hospital, London, UK (PG, AB, KM, SS, FD, UL), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London, London, UK (PG), Department of Paediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (AS), Department of Diagnostic and Interventional Neuroradiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (JF), Robert Steiner MR Facility, Medical Research Council Laboratory of Medical Sciences, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS) and the Mansfield Centre for Innovation, Imaging Sciences, Institute of Clinical Sciences, Imperial College London, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS).

From the Department of Radiology, Great Ormond Street Hospital, London, UK (PG, AB, KM, SS, FD, UL), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London, London, UK (PG), Department of Paediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (AS), Department of Diagnostic and Interventional Neuroradiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (JF), Robert Steiner MR Facility, Medical Research Council Laboratory of Medical Sciences, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS) and the Mansfield Centre for Innovation, Imaging Sciences, Institute of Clinical Sciences, Imperial College London, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS).

From the Department of Radiology, Great Ormond Street Hospital, London, UK (PG, AB, KM, SS, FD, UL), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London, London, UK (PG), Department of Paediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (AS), Department of Diagnostic and Interventional Neuroradiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (JF), Robert Steiner MR Facility, Medical Research Council Laboratory of Medical Sciences, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS) and the Mansfield Centre for Innovation, Imaging Sciences, Institute of Clinical Sciences, Imperial College London, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS).

From the Department of Radiology, Great Ormond Street Hospital, London, UK (PG, AB, KM, SS, FD, UL), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London, London, UK (PG), Department of Paediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (AS), Department of Diagnostic and Interventional Neuroradiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (JF), Robert Steiner MR Facility, Medical Research Council Laboratory of Medical Sciences, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS) and the Mansfield Centre for Innovation, Imaging Sciences, Institute of Clinical Sciences, Imperial College London, Hammersmith Hospital Campus, Du Cane Road, London, UK (JS).