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.