Enzyme Replacement Therapy for CLN2 Disease: MRI Volumetry Shows Significantly Slower Volume Loss Compared with a Natural History Cohort

Pritika Gaur; Paul Gissen; Asthik Biswas; Kshitij Mankad; Sniya Sudhakar; Felice D’Arco; Angela Schulz; Jens Fiehler; Jan Sedlacik; Ulrike Löbel

doi:10.3174/ajnr.A8408

Research ArticlePEDIATRIC NEUROIMAGING

Enzyme Replacement Therapy for CLN2 Disease: MRI Volumetry Shows Significantly Slower Volume Loss Compared with a Natural History Cohort

Pritika Gaur, Paul Gissen, Asthik Biswas, Kshitij Mankad, Sniya Sudhakar, Felice D’Arco, Angela Schulz, Jens Fiehler, Jan Sedlacik and Ulrike Löbel

American Journal of Neuroradiology October 2024, DOI: https://doi.org/10.3174/ajnr.A8408

Pritika Gaur

aFrom the Department of Radiology (P. Gaur, A.B., K.M., S.S., F.D., U.L.), Great Ormond Street Hospital, London, UK

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Paul Gissen

bNational Institute for Health Research (P. Gissen), Great Ormond Street Hospital Biomedical Research Centre, University College London, London, UK

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Asthik Biswas

aFrom the Department of Radiology (P. Gaur, A.B., K.M., S.S., F.D., U.L.), Great Ormond Street Hospital, London, UK

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Kshitij Mankad

aFrom the Department of Radiology (P. Gaur, A.B., K.M., S.S., F.D., U.L.), Great Ormond Street Hospital, London, UK

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Sniya Sudhakar

aFrom the Department of Radiology (P. Gaur, A.B., K.M., S.S., F.D., U.L.), Great Ormond Street Hospital, London, UK

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Felice D’Arco

aFrom the Department of Radiology (P. Gaur, A.B., K.M., S.S., F.D., U.L.), Great Ormond Street Hospital, London, UK

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Angela Schulz

cDepartment of Paediatrics (A.S.), University Medical Center Hamburg-Eppendorf, Hamburg, Germany

gGerman Center for Child and Adolescent Health (A.S.), Partner Site Hamburg, Hamburg, Germany

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Jens Fiehler

dDepartment of Diagnostic and Interventional Neuroradiology (J.F.), University Medical Center Hamburg-Eppendorf, Hamburg, Germany

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Jan Sedlacik

eRobert Steiner MR Facility (J.S.), Medical Research Council Laboratory of Medical Sciences, Hammersmith Hospital Campus, London, UK

fMansfield Centre for Innovation (J.S.), Imaging Sciences, Institute of Clinical Sciences, Imperial College London, Hammersmith Hospital Campus, London, UK

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Ulrike Löbel

aFrom the Department of Radiology (P. Gaur, A.B., K.M., S.S., F.D., U.L.), Great Ormond Street Hospital, London, UK

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Enzyme Replacement Therapy for CLN2 Disease: MRI Volumetry Shows Significantly Slower Volume Loss Compared with a Natural History Cohort

Pritika Gaur, Paul Gissen, Asthik Biswas, Kshitij Mankad, Sniya Sudhakar, Felice D’Arco, Angela Schulz, Jens Fiehler, Jan Sedlacik, Ulrike Löbel

American Journal of Neuroradiology Oct 2024, DOI: 10.3174/ajnr.A8408

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Enzyme Replacement Therapy for CLN2 Disease: MRI Volumetry Shows Significantly Slower Volume Loss Compared with a Natural History Cohort

Pritika Gaur, Paul Gissen, Asthik Biswas, Kshitij Mankad, Sniya Sudhakar, Felice D’Arco, Angela Schulz, Jens Fiehler, Jan Sedlacik, Ulrike Löbel

American Journal of Neuroradiology Oct 2024, DOI: 10.3174/ajnr.A8408

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Enzyme Replacement Therapy for CLN2 Disease: MRI Volumetry Shows Significantly Slower Volume Loss Compared with a Natural History Cohort

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