A new DRUG TRIALS SNAPSHOT is now available.
BRINEURA is a drug that slows loss of walking ability (ambulation) in symptomatic patients with a specific form of Batten disease. It is to be used in patients 3 years of age and older.
This form of Batten disease is known as late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). CLN2 is a rare, inherited, childhood disease that is characterized by the deficiency of tripeptidyl peptidase-1 (TPP1) enzyme, primarily affecting the central nervous system. The lack of enzyme causes progressive problems with vision, movement, and thinking ability and premature death.