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News From the Food and Drug Administration
June 6, 2017

Therapy for Rare Nerve Cell Disease

JAMA. 2017;317(21):2157. doi:10.1001/jama.2017.6334

The FDA has approved the first treatment for a rare form of Batten disease in which initial symptoms including language delay, epilepsy, and ataxia affect young children, usually at age 2 to 4 years.

Cerliponase alfa, marketed as Brineura, was approved to slow ambulation loss in symptomatic children aged 3 years or older who have late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidal peptidase-1 (TPP1) deficiency. Neuronal ceroid lipofuscinoses are rare, inherited neurodegenerative disorders known collectively as Batten disease.