Real-world clinical outcomes of patients with CLN2 disease treated with cerliponase alfa

Real-world clinical outcomes of patients with CLN2 disease treated with cerliponase alfa

IntroductionThis study assessed the real-world effectiveness and safety of the enzyme replacement therapy (ERT), cerliponase alfa, to treat neuronal ceroid lipofuscinosis type 2 (CLN2) disease.MethodsData from the DEM-CHILD database were analyzed, comparing patients who initiated ERT outside clinica...

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Main Authors: Angela Schulz, Christoph Schwering, Eva Wibbeler, Lena Marie Westermann, Luca Hagenah, Susanne Lezius, Ashok Jha, Abigail Hunt, Peter Slasor, Pascal Reisewitz, Miriam Nickel
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-03-01
Series:Frontiers in Neurology
Subjects:
cerliponase alfa
CLN2
effectiveness
safety
real-world data
DEM-CHILD
Online Access:https://www.frontiersin.org/articles/10.3389/fneur.2025.1516026/full
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https://www.frontiersin.org/articles/10.3389/fneur.2025.1516026/full

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