Shots:

• The study involves assessing Brineura (cerliponase alfa- 300mg) in 24 patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease- aged 3-16 yrs. with motor-language (ML) score 3-6 on CLN2 clinical rating scale- conducted from Sep-2013 to Nov-2015 in the US- including Germany and Italy and two in the UK
• The study demonstrated durability in treatment with 83% with ML score @3yrs. & @2yrs were 3.8 & 3.3 points
• Brineura (cerliponase alfa) is an enzyme replacement therapy for restoring TPP1 enzyme deficient in patients with CLN2 disease administered directly into CSF in brain and has received the US FDA & EU approval in April-17 and June-17 for late infantile neuronal CLN2 respectively

Ref: PRNewswire | Image: Biomarin