Clinical Edge

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Cerliponase Alfa Impresses for CLN2 Disease

Key clinical point: Children with a form of Batten disease treated via intraventricular infusion of cerliponase alfa for 3 years showed significantly less decline in motor and language function.

Major finding: A positive treatment response was seen in 83% of patients after 3 years.

Study details: This is an ongoing, open-label study of 23 children with ceroid lipofuscinosis type 2 disease treated with cerliponase alfa.

Disclosures: The study was funded by BioMarin Pharmaceutical, which markets Brineura. Dr. Trivisano was a subinvestigator in the trial.


Trivisano M et al. IEC 2019, Abstract P333.