Study of Intraventricular Cerliponase Alfa for CLN2 Disease

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Abstract

<p class="first" id="d5646802e137">Recombinant human tripeptidyl peptidase 1 (cerliponase alfa) is an enzyme-replacement therapy that has been developed to treat neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a rare lysosomal disorder that causes progressive dementia in children. </p>

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Journal

Title: New England Journal of Medicine

Abbreviated Title: N Engl J Med

Publisher: New England Journal of Medicine (NEJM/MMS)

ISSN (Print): 0028-4793

ISSN (Electronic): 1533-4406

Publication date Created: April 24 2018

Publication date (Electronic): April 24 2018

Affiliations

[1 ]From the Department of Pediatrics, University Medical Center Hamburg–Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Gesù Children’s Hospital, IRCCS, Rome (N.S.); Nationwide Children’s Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill Cornell...

Article

DOI: 10.1056/NEJMoa1712649

SO-VID: b06fdbcc-6622-4385-b15e-c80f10e2d967

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