BioMarin Reports Results of Brineura (cerliponase alfa) for Patients with CLN2 Disease Aged 3 to 16 yrs.

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

Click here to read full press release/ article | Ref: PRNewswire | Image: Biomarin