FDA approves first treatment for a form of Batten disease

By Newsroom America Feeds at 27 Apr 2017

The U.S. Food and Drug Administration today approved Brineura (cerliponase alfa) as a treatment for a specific form of Batten disease. Brineura is the first FDA-approved treatment to slow loss of walking ability (ambulation) in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm555613.htm

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