Study of Intraventricular Cerliponase Alfa for CLN2 Disease

2018472 citationsJournal Article

Authors

Angela Schulz · University Medical Center Hamburg-Eppendorf

Temitayo Ajayi · BioMarin (United States)

Nicola Specchio · Bambino Gesù Children's Hospital

Emily de los Reyes · Nationwide Children's Hospital

Paul Gissen · Great Ormond Street Hospital

Douglas Ballon · Citigroup

Jonathan P. Dyke

Abstract

Intraventricular infusion of cerliponase alfa in patients with CLN2 disease resulted in less decline in motor and language function than that in historical controls. Serious adverse events included failure of the intraventricular device and device-related infections. (Funded by BioMarin Pharmaceutical and others; CLN2 ClinicalTrials.gov numbers, NCT01907087 and NCT02485899 .).

Topics & Keywords

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Trypanosoma species research and implications

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: New England Journal of Medicine

Volume 378, Issue 20, pp. 1898-1907

DOI: 10.1056/nejmoa1712649

Field-Weighted Citation Impact: 31.50