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Late infantile neuronal ceroid lipofuscinosis 2

MONDO:0979344

Also known as: late infantile CLN2 disease

2 clinical trials for this condition and its sub-types.

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Broader categories

Disease (680) Metabolic disease (233) Nervous system disorder (231) Inherited lipid metabolism disorder (189) Hereditary disease (176) Neurodegenerative disease (171) Central nervous system disorder (107) Inborn errors of metabolism (45) Lysosomal storage disease (35) Human disease (14)

Trials to join now! 1 Not yet finished but already full! 1

Rare disease drug safety tracked in new observational study

Knowledge-focused Ongoing

This study follows 35 children with CLN2 disease (a rare, severe brain disorder) who are taking cerliponase alfa (Brineura). Researchers will monitor for side effects and serious reactions over time. The goal is to better understand the long-term safety of this enzyme replacement…

Sponsor: BioMarin Pharmaceutical • Aim: Knowledge-focused

Last updated Jul 02, 2026 00:00 UTC