Persistent Treatment Effect of Cerliponase Alfa in Children with CLN2 Disease: A 3 Year Update from an Ongoing Multicenter Extension Study

• DOI: 10.1055/s-0039-1698182
• Publication Date: 2019-09-30T18:18:44Z
• Authors: Angela Schulz; Nicola Specchio; Paul Gissen; Emily de los Reyes; Heather Cahan; Peter Slasor; David Jacoby; Temitayo Ajayi
• Keywords: 0301 basic medicine; 03 medical and health sciences; 0302 clinical medicine

Background: CLN2 disease, a rare, inherited, pediatric, neurodegenerative lysosomal storage disorder caused by TPP1 deficiency, is characterized seizures, language and motor function loss, blindness, early death. An open-label study demonstrated that intracerebroventricular (ICV) infusion of 300 mg cerliponase alfa, recombinant human enzyme, every other week for 48 weeks slowed deterioration in function. This extension (NCT02485899) assesses the long-term safety efficacy alfa up to 240 weeks.