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What condition is Brineura used to treat?

Medically reviewed by Last updated on June 25, 2020.

Official Answer


Brineura (cerliponase alfa) works as a treatment for a specific form of Batten disease, and is the first FDA-approved treatment that works to slow down the loss of the ability to walk (ambulation) in pediatric patients ages 3 years or older. Batten disease is a rare and fatal genetic neurodegenerative disorder that begins in childhood. Brineura replaces TPP1, an enzyme deficient in patients that have late infantile neurotic ceroid lipofuscinosis type 2 (CLN2).

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